Lentiviral vector gene therapy. There are two types of vectors, viral and non-viral.

Lentiviral vector gene therapy Growing momentum Apr 15, 2000 · Retroviral vectors derived from murine retroviruses are being used in several clinical gene therapy trials. (Funded by Rocket Pharmaceuticals and the California Institute for Regenerative Oct 14, 2022 · After years of delays and setbacks, the lentiviral vector gene therapy platform has secured two FDA approvals. Although existing data increasingly support the usefulness and safety of clinical-grade lentiviral vectors used in cell The concept of gene therapy originated in the mid twentieth century and was perceived as a revolutionary technology with the promise to cure almost any disease of which the molecular basis was understood. clinicaltrials. Gene therapy works by introducing genetic material (such as a transgene or nuclease) into the nucleus of the cell. This review presents the state of the art of the production of these vectors with particular emphasis on their large-scale production for clinical purposes. This Aug 1, 2020 · Lentiviral vectors (LVs) have provided an efficient way to integrate our gene of interest into eukaryote cells. Examples are Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. The use of lentiviral vectors in cell and gene therapy is steadily increasing, both in commercial and investigational therapies. This Sep 14, 2023 · Lentiviral (LV) vectors are important gene-delivery tools for cell and gene therapy applications. With the extensive and in-depth studies on this gene therapy vehicle over the past two decades, LVs have been widely … Lentivirus is a genus of the Retroviridae family, characterized by a long incubation period. Aug 2, 2021 · Abstract Lentiviral vectors are the most frequently used tool to stably transfer and express genes in the context of gene therapy for monogenic diseases. To facilitate LV design, optimize transduction, and assess potential therapeutic efficacy, we leveraged a human cellular model of DBAS based on heterozygous disruption of RPS19 in healthy donor CD34 + HSPCs. Third-generation, self-inactivating Jun 12, 2025 · Using a γ-globin lentivirus vector with and without a small insulator, Malik and colleagues show both vectors correct sickle cell anemia in mice and are safe in CRISPR-targeting LMO2 assay. Discover the challenges faced in this process and the innovative solutions being developed in the field. Explore lentiviral vector-based process solutions to simplify progress in every step of your lentivirus gene therapy workflow. Jul 6, 2020 · We explored two parameters for improving gene transfer efficacy and capacity for the selective targeting of subpopulations of cells with lentiviral vectors (LVs). May 19, 2016 · Lentiviral vectors are one of the most popular and useful viral vectors in the lab. Performed in collaboration with CSL Behring, this study explores the use of cryo-transmission electron microscopy (cryo-TEM) for the detailed characterization of lentivirus-based vectors, aiming to enhance our understanding and optimization of these critical therapeutic tools. Lentiviruses can deliver a significant amount of genetic information into the DNA of the host cell, so they are one of the most efficient methods of a gene delivery vector. Examples are Aug 2, 2021 · Lentiviral vectors are the most frequently used tool to stably transfer and express genes in the context of gene therapy for monogenic diseases. Mar 14, 2024 · Located at the Center for Cellular Immunotherapies (CCI), the innovation center for many novel gene therapy products including tisagenlecleucel (Kymriah), the authors were uniquely positioned to evaluate the stability of 13 clinical-grade LVs produced during the past decade. Tisagenlecleucel (Kymriah), axicabtagene ciloleucel (Yescarta) and most recently brexucabtagene autoleucel (Tecartus) are examples of T cell therapies which are now commercially available for distribution after successfully obtaining EMA and FDA approval for Lentivirus as a Vector in Gene Therapy Advantages of Utilizing Lentiviral Vectors in Gene Therapy: Lentiviral vectors offer a relatively large packaging capacity, which is advantageous for accommodating larger genetic payloads compared to vectors like adeno-associated viruses. The Lenti/G-βAS3-FB lentiviral vector was designed to express a modified β-globin gene (βAS3) that produces an anti-sickling hemoglobin. reported an interim analysis of a single-center, single-arm pilot trial evaluating a β-globin expression-optimized and insulator-engineered lentivirus-modified cell product in the most severe β-thalassemia (β0/β0). This comprehensive analysis demonstrates long-term stability by titer, product transduction efficiency, and potency. Introduction Viral vectors have been derived from a large number of viruses, which were transformed into efficient tools for delivery of genes into cells of interest, thanks to their unique characteristics. A third-generation lentiviral vector pseudotyped with Sendai virus F and HN envelope proteins (rSIV. Vectors are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell. This therapy is well tolerated and provides stable long-term expression of FAH in pigs with HT1. For clinical use, these viral vectors must be generated under conditions of good manufacturing practice in large quantities, which In this work, we present an optimized approach to produce LV encoding the CD19 gene of interest, benchmarked against a green fluorescent protein (GFP) control. gov). An extensive analysis of vector insertion sites thus far shows a highly polyclonal hematopoiesis by the transduced cells. In Ada−/− mice, neonatal treatment resulted in broad vector marking across all tissues analyzed, whereas adult treatment Oct 9, 2024 · Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti-D) has shown efficacy in Mar 13, 2020 · Adenosine deaminase (ADA)-deficient mice and healthy rhesus monkeys were studied to determine the impact of age at treatment, vector dosage, dosing schedule, repeat administration, biodistribution, and immunogenicity after systemic delivery of lentiviral vectors (LVs). Methods and applications involving lentiviral production are frequently changing. Apr 15, 2000 · Retroviral vectors derived from murine retroviruses are being used in several clinical gene therapy trials. (Funded by Bluebird … Sep 9, 2016 · Lentiviral vectors could also be used to generate transgenic mice in which the expression of a specific gene was reduced by vector-encoded small-hairpin RNAs (9). These advantages include a larger genetic cargo capacity, efficient transduction of both dividing and non-dividing cells, stable integration into the host genome for long-term expression, and relatively low Aug 25, 2022 · Indeed, the phenotypic and genomic data following in vivo lentiviral vector administration demonstrate comparative superiority over other therapies including ex vivo cell therapy and therefore Oct 9, 2024 · Conclusions At a median follow-up of 6 years after lentiviral gene therapy, most patients with early cerebral adrenoleukodystrophy and MRI abnormalities had no major functional disabilities. Lentiviral vectors (LV) have seen considerably increase in use as gene therapy vectors for the treatment of acquired and inherited diseases. developed novel CD90-targeted viral vectors to selectively transduce HSCs with enhanced on-target efficiency to improve the feasibility and safety of ex vivo gene therapy. Over the past two decades, increasing studies have shown that lentiviral vectors (LVs) modified with different Jan 24, 2022 · The final report of the phase 1/2 lentiviral gene therapy trial in severe β-hemoglobinopathies, HGB-205, shows a long-term safety profile and sustained reduction of transfusion requirements in From these vectors, one of the most widely used virus vector is the one based on the human immunodeficiency virus 1 (HIV-1) lentivirus. May 3, 2010 · It seems reasonable to conclude for now that we need results from additional patients treated with lentiviral vector gene transfer before we can draw solid conclusions regarding the relative safety of this vector system. Cells were isolated from the tracheas of rats and used to establish air-liquid interface (ALI) cultures. Jul 5, 2024 · Li et al. Feb 20, 2025 · BI 3720931 is a first-in-class, inhaled lentiviral vector-based gene therapy that could potentially address unmet needs by inserting a functional copy of the CFTR gene in the DNA of airway Dec 21, 2022 · Lentiviral Gene Therapy for Artemis-Deficient SCID Results Marrow harvest, busulfan conditioning, and lentiviral-transduced CD34+ cell infusion produced the expected grade 3 or 4 adverse events. Direct modification of CD34+CD90+ HSCs with targeted vectors further paves the way for future in vivo HSC gene therapy applications. Dec 18, 2024 · The design of this lentiviral vector raises unique safety risks, because expression of the therapeutic transgene is driven by a potent murine γ-retroviral enhancer–promoter that can efficiently Dec 12, 2022 · Disclosed in the present invention is a lentiviral vector applicable to gene therapy of thalassemia and sickle anemia, comprising: a, a micro locus control region, which is a micro regulatory element that is screened from a locus control region of beta globin 16kb and does not contain an HS1 region; b, a gene sequence of beta globin, wherein the gene sequence is as shown in SEQ ID NO. This treatment involves the collection of patients’ stem cells, ex vivo gene transfer using the Lenti/G-βAS3-FB vector, and subsequent myeloablative marrow conditioning with busulfan prior to the re-administration of the gene-modified stem cells. These vectors incorporate features to provide long-term gene transfer and expression while minimizing generation of a replication-competent virus or pathogenicity. However, with their clinical application, potential safety issues have Lentiviral vectors (LVs), derived from human immunodeficiency virus, are powerful tools for modifying the genes of eukaryotic cells such as hematopoietic stem cells and neural cells. Mar 14, 2024 · As the use of GMP-grade lentiviral vectors for clinical cell therapy manufacturing increases, the high cost, limited production facilities, and long queues necessitate that vector lots remain stable in long-term storage for extended use. Shaw, PhD, Elizabeth Garabedian, RN, MS, Oct 10, 2024 · At a median follow-up of 6 years after lentiviral gene therapy, most patients with early cerebral adrenoleukodystrophy and MRI abnormalities had no major functional disabilities. One of the major challenges for an effective gene therapy approach is the design of an efficient vector that combines high-level and long-term β-globin expression with high infectivity in primary CD34+ cells. Through a carefully orchestrated interplay of these genes, the packaging plasmid ensures that the lentiviral vector's genetic cargo is accurately and efficiently assembled into new virus particles. Abstract Lentiviral vectors are among the most used vectors in gene therapy to treat pathologies of different origins, such as cancers, rare monogenic diseases or neurological disorders. This guideline describes quality aspects and non-clinical testing that are in general relevant for LV that are intended for ex vivo or in vivo Mar 1, 2021 · Lentiviral vector bioprocess economics for cell and gene therapy commercialization Ruxandra-Maria Comisel a , Bo Kara b , Frederick H. Think of a vector as a microscopic delivery truck that transports packages (genetic material) to specific locations (target cells). This abled LVs to be used in both research laboratories and gene therapy. Keywords: hematopoietic stem cells; hemophilia A; lentiviral gene therapy. Recently, progress has been made in the development of vectors based on the lentivirus genus of retroviruses, which ironically includes a major human pathogen, human immunodeficiency virus (HIV). Abstract Viral vectors provide an efficient means for modification of eukaryotic cells, and their use is now commonplace in academic laboratories and industry for both research and clinical gene therapy applications. Jul 30, 1999 · Although lentiviral vectors hold promise as delivery vehicles for gene therapy, their parental origin has raised considerable safety concerns that have so far precluded human testing. Since then, several gene vectors have been developed and the feasibility of gene therapy has be … Lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy, using an insulin-like growth factor 2 tag with R37A mutein, demonstrated long-term reversal of pathological effects in central nervous system, muscles, and improvement of locomotor function, including safety by integrations site assessment and single-cell RNA-sequencing analysis of brain. Among the various tools used for gene delivery, lentiviral vectors have emerged as a popular choice due to their ability to stably integrate into the host genome and efficiently transduce both dividing and non-dividing cells. Jul 14, 2021 · Lentiviral vectors (LVs), derived from human immunodeficiency virus, are powerful tools for modifying the genes of eukaryotic cells such as hematopoietic stem cells and neural cells. Dec 13, 2024 · Bhoopalan and colleagues have developed a lentiviral vector encoding RPS19, the most commonly mutated gene in patients with Diamond-Blackfan anemia syndrome (DBAS) and demonstrated the safety and efficacy of this vector for gene therapy using different cell models. Abstract Three gene therapy strategies have received US Food and Drug Administration (FDA) approval; one includes HIV-1-based lentiviral vectors. LVs produced in most research laboratories contain contaminants that can generate confounding effects in experimental studies. Lentiviral vectors in gene therapy is a method by which genes can be inserted, modified, or deleted in organisms using lentiviruses. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. 3% of patients developed second primary malignancies, and vector integration analyses revealed Abstract Lentiviral vectors (LVs) have gained value over recent years as gene carriers in gene therapy. The state of the art of vector production will be summarized face to Aug 18, 2023 · Additionally, the packaging plasmid often contains the Rev gene, responsible for regulating the nuclear export of viral RNA transcripts. Oct 4, 2023 · Kiem et al. Apr 26, 2025 · However, lentiviral vectors, particularly those produced from stable lentiviral packaging cell lines, have played a huge role in increasing accessibility to gene therapy in a clinical setting and Jul 24, 2025 · These viral vector strategies rely on adenoviruses, adeno-associated viruses, lentivirus-derived tools, and so on. Learn how lentiviral vectors, derived from HIV-1, are used in immuno-oncology and liver-directed gene therapy. A preclinical study conducted by researchers at Children’s Hospital of Philadelphia (PA, USA) has demonstrated an improved gene therapy approach for metachromatic leukodystrophy (MLD), a rare, progressive pediatric neurological disease. HIV, SIV, and FIV are all examples of lentiviruses. 13 However, in clinical trials involving Apr 27, 2020 · Autologous gene therapy using lentiviral vectors (LVs) holds promise for treating monogenetic blood diseases. This characteristic makes LVs ideal for clinical research, as has been demonstrated with the approval of Keywords: lentiviral vector, gene therapy, T cell, B cell, HSC, CRISPR/CAS9, NK cell, pseudotyping 1. Scientists use the existing structure and mechanisms of the Jul 31, 2022 · This review offers the basics of lentiviral vector technologies, their advantages and pitfalls, and an overview of their use in the field of ophthalmology. Lentiviral vectors are also being tried for the gene therapy of various other genetic and acquired diseases, by administration into the eye or brain, or intravenously to reach the liver. As these vector systems for clinical gene transfer are developed, it is important to The aim of this study was to employ two CF rat models, Phe508del and CFTR knockout (KO), to assess the comparative effectiveness of CFTR modulators and lentiviral (LV) vector-mediated gene therapy. Sep 1, 2023 · Gene therapy holds great promise for curing cancer by editing the deleterious genes of tumor cells, but the lack of vector systems for efficient delivery of genetic material into specific tumor sites in vivo has limited its full therapeutic potential in cancer gene therapy. Feb 13, 2025 · Lentiviral vectors for gene therapy of α-thalassemia Letter to the Editor Published: 13 February 2025 Volume 68, pages 2177–2179, (2025) Cite this article Nov 29, 2002 · What do lentiviral vectors newly offer to the field of gene therapy? Unlike other retroviruses, lentivectors do not necessarily require cell division for proviral integration and productive Treated mice displayed sustained plasma fVIII levels and no signs of product related toxicity. The risks and the measures taken to minimize secondary effects as well as new Many of the appropriate quality and safety requirements for retroviral vectors have been described in the current Note for Guidance (NfG) on the quality, preclinical and clinical aspects of gene transfer medicinal products, CPMP/BWP/3088/99). Learn more about comparison with AAVs and how lentiviral vector manufacturing challenges – from plasmid design to aseptic filling and controlled freezing – can be solved to enhance overall manufacturing efficiency. Jan 24, 2022 · Long-term monitoring of patients with Wiskott–Aldrich syndrome following lentiviral gene therapy shows a safe profile and a reduction in the frequency of autoimmune manifestations and bleeding Jan 24, 2022 · Long-term monitoring of patients with Wiskott–Aldrich syndrome following lentiviral gene therapy shows a safe profile and a reduction in the frequency of autoimmune manifestations and bleeding Mutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (CF), irrespective of their CF transmembrane conductance regulator (CFTR) gene variants. Sep 6, 2025 · The team’s innovative enhancement revolves around optimizing both the lentiviral vector design and in vivo delivery protocols to circumvent these limitations. As these vector systems for clinical gene transfer are developed, it is important to Abstract Lentiviral vectors have played a critical role in the emergence of gene-modified cell therapies, specifically T cell therapies. Keywords: lentiviral vector, gene therapy, rhesus monkeys, in vivo, immune response, ADA-deficiency, ERT, repeat administration Introduction Feb 24, 2009 · Lentiviral vectors (LVs) have become some of the most widely used vectors for fundamental biological research, functional genomics, and gene therapy. The agency approved bluebird bio’s betibeglogene autotemcel for severe β Creative Biolabs offers diverse Lentiviral vector design and construction used as delivery systems for gene therapy. Jul 7, 2005 · Progress in the development of serum-free media for clinical grade retroviral vector production will continue. First, the description of the global challenges encountered to develop safe and efficient lentiviral recombinant vectors for clinical applicatio … Aug 25, 2022 · Here we demonstrate cure of HT1 by direct, in vivo administration of a therapeutic lentiviral vector targeting the expression of a human fumarylacetoacetate hydrolase (FAH) transgene in the porcine model of HT1. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; lovotibeglogene autotemcel) consis Dec 15, 2024 · A third-generation lentiviral vector pseudotyped with Sendai virus F and HN envelope proteins (rSIV. Kohn, MD, Kit L. Brusson et al. Examples are hematopoietic stem Jun 5, 2025 · Lentivirus-based vectors have emerged as a powerful tool for therapeutic gene delivery, playing a critical role in the approval of several cell and gene therapies. May 9, 2025 · Gene therapy has rapidly evolved as a promising approach for treating a wide range of genetic disorders. The vast majority of clinical applications involves an ex vivo modality whereby lentiviral vectors are used to transduce autologous somatic cells, obtained from patients and re-delivered to patients after transduction. Dec 12, 2021 · Sickle cell disease is characterized by the painful recurrence of vaso-occlusive events. Abstract Lentiviral vectors are increasingly the gene transfer tool of choice for gene or cell therapies, with multiple clinical investigations showing promise for this viral vector in terms of both safety and efficacy. Human immunodeficiency virus (HIV)-derived LVs have been vastly studied to become an invaluable asset in gene delivery. These viral vectors are safer than what was previously being used for gene transfer and are capable of infecting both dividing and nondividing cells with a long-term expression. Apr 17, 2019 · Lentiviral vector gene therapy combined with low-exposure, targeted busulfan conditioning in infants with newly diagnosed SCID-X1 had low-grade acute toxic effects and resulted in multilineage Pseudotyped lentiviral vectors are common in clinical applications, like gene therapy, as you can more specifically dictate the target cell population and reduce cellular toxicity. Jan 22, 2022 · Contrarily, haematopoietic stem and progenitor cell gene therapy (HSPC-GT) consisting of an autologous transplant of haematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a lentiviral vector encoding the ARSA gene, resulted in prevention and correction of CNS and peripheral nervous system in the mouse model. Lentiviral vectors are derived from viruses that integrate the transgene into the DNA genome of the target cells. This chapter provides an overview on lentiviral vector developments in terms of vector design and manufacture for gene therapy applications. Importantly, despite the use of a lentiviral vector that contained an internal viral LTR to drive transgene expression, no clustering of vector insertions in oncogenes or growth-related genes was observed. The system used to deliver genetic material is known as a vector. Oct 1, 2025 · Here, we describe a clinical candidate developed with a platform enabling transduction of targeted cell types in vivo utilizing an engineered, third-generation lentiviral vector with rationally designed fusogen and binder moieties that enable the vector to target specific cells (Figure 1 B). Current LVLP systems predominantly utilize HIV-Gag and Pol proteins Dec 11, 2020 · Lentiviral vectors (LVs) are powerful gene-transfer tools routinely exploited for distinct research and clinical applications. The first clinical trial with a lentiviral vector is underway with others anticipated. Promising preclinical results support the progression of this vector carrying a full-length CFTR transgene (BI 3720931) into a first-in-human clinical trial expected to begin in 2024. Lentiviruses are a family of viruses that are responsible for diseases like AIDS, which infect by inserting DNA into their host cells' genome. Lentiviral vectors, the type of vector that Orchard uses, are particularly well-suited for our HSC gene therapy approach. An LV driving the expression of interferon-alpha (IFNα) is injected intravenously in the tail vein of an immunocompetent mouse-bearing established LMS. These can infect both dividing and non-dividing cells, allowing for stable transgene expression in various cell types Dec 9, 2024 · Gene therapy for hemophilia A with the use of lentiviral vector–transduced autologous HSCs resulted in stable factor VIII expression, with factor VIII activity correlating to vector copy number Lentivirus is a genus of the Retroviridae family, characterized by a long incubation period. Collectively, the findings of the current study support the preclinical safety and efficacy of CD68-ET3-LV CD34 +. First, the description of the global challenges encountered to develop safe and efficient lentiviral recombinant vectors for clinical application is provided. However, depending on the specific application, difficulties have persisted with respect to the efficiency and safety of this vector system The main interest of viral vectors for gene therapy purposes lays in the first, collectively referred to as the early steps of the viral life cycle. Soldi et al. Apr 4, 2025 · Background The development of gene editing tools such as CRISPR-Cas9 and base editors (BE) is critical for genetic diseases and cancer. However, in contrast to γ-RV, LV can also transduce nondividing cells. LVs hold notable and inherent advantages over other gene transfer agents based on their ability t … Feb 9, 2010 · This first success, achieved after retroviral therapy, was later overshadowed by the occurrence of vector-related leukemia in a significant number of the treated children, demonstrating that the future success of gene therapy depends on our understanding of vector biology. With the extensive and in-depth studies on this gene therapy vehicle over the past two decades, LVs have been widely used in both research and clinical trials. From the middle of the gene therapy transition, despite the challenges and serious negative consequences, the lentiviral vector has emerged as a cornerstone and demonstrated benefits in fields ranging from basic science to gene Since their development in the 1990s, the applications of lentiviral vectors have rapidly extended to the fields of gene and cancer therapy, as well as vaccine development, owing to their unique advantages of efficiently transducing both dividing and nondividing cells and achieving stable and long-t … Mar 13, 2020 · Adenosine deaminase (ADA)-deficient mice and healthy rhesus monkeys were studied to determine the impact of age at treatment, vector dosage, dosing schedule, repeat administration, biodistribution, and immunogenicity after systemic delivery of lentiviral vectors (LVs). Lentiviral vectors, derived from the human immunodeficiency virus, have been extensively investigated and optimized over the past two decades. Approved therapies now using LV vectors include Zynteglo for beta-thalassemia and Skysona for cerebral adrenoleukodystrophy. The team What is lentiviral vector transduction? Lentiviral vector transduction refers to the use of modified lentiviruses to perform gene editing in cell and gene therapies. Discover how researchers overcome systemic delivery challenges and improve biodistribution and safety of lentiviral vectors. The concept of gene therapy originated in the mid 20th century and was perceived as a revolutionary technology with the promise to cure almost any disease of which the molecular basis was understood. This process can be defined as gene transduction (in gene therapy) or single cycle infection (in virology) and both terms will be used here. One common way that researchers have found to accomplish this is by using a vector. Introduction to Lentiviral Vectors The field of gene and cell therapy is advancing rapidly with lentiviral-based vectors being the preferred vector of choice due to their ability to infect both dividing and non-dividing cells and integrate transgenes into the host cell genome [1]. Mar 18, 2025 · An approved gene therapy has been modified with a new lentiviral vector, demonstrating improved safety and expanding the eligibility criteria. However, clinical applications can be limited by suboptimal hematopoietic stem cell These studies identify important factors to consider related to age and timing of administration when implementing systemic delivery of LVs as a potential therapeutic agent. describe a laboratory-scale workflow for purified LV production, highlighting enhanced gene-editing efficiency and diminished Oct 24, 2024 · Our research, which could be a game-changer, hypothesizes that ex vivo lentiviral (LV) gene therapy (GT) could produce the supraphysiological level of active GALNS enzyme by hematopoietic stem cells (HSCs) transduced with LVs carrying the native GALNS gene under two different promoters (CBh and COL2A1), impacting bone and cartilage Gene therapy is a promising therapeutic alternative, particularly in patients lacking an allogeneic bone marrow (BM) donor. This review offers the basics of lentiviral vector technologies, their advantages and pitfalls, and an overview of their use in the field of ophthalmology. First, the description of the global challenges encountered to develop safe and efficient May 11, 2021 · An alternative treatment, ex vivo autologous hematopoietic stem-cell gene therapy with a γ-retroviral vector, 11,12 was approved in Europe in 2016. Lentiviral vectors are of particular interest because of their favorable biosafety profile and ability to introduce their therapeutic cargo into non-dividing cells. LV resembles γ-retroviral vectors (γ-RVs) in their ability to stably integrate into the target cell genome, resulting in persistent expression of the gene of interest. For instance, third-generation and self-inactive LVs Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. Mar 22, 2018 · Although the use of gammaretroviral vectors is more common, especially in the research setting, the number of clinical trials using lentiviral vectors for gene therapy is increasing. Third-generation, self-inactivating Jan 30, 2024 · Viral Vectors Gene therapy is a way to treat or prevent a specific disease using genetic material. developed a lentiviral vector expressing an anti-sickling β-globin and an artificial microRNA downregulating βS-globin expression; this vector reduces HbS levels and effectively corrects the sickling phenotype. We would like to show you a description here but the site won’t allow us. Reliable methods to characterize lentiviral vectors and the cells transduced with them are critical to helping ensure the safety and efficacy of these biologics. However, insertional oncogenesis is an ongoing risk associated with the integration of viral vectors. Viral vectors are currently a Nov 17, 2025 · Explore the latest advances in R&D and manufacturing of in vivo lentiviral vectors. 1. Read on to learn more about the current Jul 31, 2022 · This review offers the basics of lentiviral vector technologies, their advantages and pitfalls, and an overview of their use in the field of ophthalmology. Advantages of lentivirus include a large genetic capacity and the ability to transduce both dividing and non-dividing cells. Since then, several gene vectors have been Jul 5, 2025 · Since their development in the 1990s, the applications of lentiviral vectors have rapidly extended to the fields of gene and cancer therapy, as well as vaccine development, owing to their unique advantages of efficiently transducing both dividing and nondividing cells and achieving stable and long-term expression of genes. Jul 27, 2021 · The viral vector used is largely dependent on the disease and cell type that needs correcting. A Lentivirus Vector is a type of retrovirus that can deliver genes into both dividing and nondividing cells by passing through the nucleus membrane, making it a valuable tool for gene therapy in various tissues, including the central nervous system and retinal cells. Nov 17, 2025 · Gene therapy has become a widely accepted treatment for inherited or acquired genetic diseases. Jan 17, 2024 · Schematics showing lentiviral vector (LV)-based gene therapy of liver metastases (LMS) in mice. F/HN) has been developed for the treatment of CF. Our process achieved high upstream LV titers while consistently maintaining an infectivity-to-particle ratio below 1:1000. Apr 30, 2025 · In this study, lentiviral vector–transduced autologous CD34+ HSCT was successful in treating severe LAD-I. Mar 22, 2023 · Sickle cell disease is due to a mutation in the β-globin gene causing production of the toxic sickle hemoglobin. The aim of this study was to employ two Nov 13, 2019 · Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort Donald B. Fiesser c , Suzanne S. There are two types of vectors, viral and non-viral. 3; c, a Jan 1, 2015 · This chapter discusses lentiviral vectors properties and applications in gene therapy for neurodegenerative diseases, presenting some of the recent progress in this field. The third-generation vector system is well characterized, effectively delivers genetic material and maintains long-term stable expression in target cells, delivers larger May 23, 2025 · In vivo gene therapy to the liver using lentiviral vectors (LV) may represent a one-and-done therapeutic approach for monogenic diseases. Farid a Show more Add to Mendeley Jul 2, 2025 · We developed a self-inactivating third-generation RPS19-encoding lentiviral vector (LV) called SJEFS-S19 for DBAS gene therapy. We also present the materials and methods necessary to generate high-titer lentiviral vectors. Lentiviral vectors are the vector of choice for many CRISPR applications, and they’ve also had success in clinical gene therapy applications. Chief among these is the possibility that a lentivirus that is able to self-replicate could be produced during manufacture of the vector in the packaging cell line or in the target cells by a process of An extensive analysis of vector insertion sites thus far shows a highly polyclonal hematopoiesis by the transduced cells. Most often, lentiviral vectors are used in gene-modified cell therapies, where cells are modified ex vivo and then injected into patients to deliver the therapy. Lentivirus-like particles (LVLPs) grows into an auspicious platform for delivering mRNA or ribonucleic proteins (RNPs) due to it integrates the advantage of viral and non-viral vectors. However, as with any medical intervention, the use of lentiviral . Jan 20, 2025 · In an analysis of long-term safety events in 783 patients treated with T cell therapy in 38 trials, 2. Increasing LV gene therapy potency is crucial for reducing Lentiviral vectors (LV) are increasingly favored for both in vivo and ex vivo gene therapy due to their distinct advantages over other viral vectors such as adeno-associated virus (AAV). In contrast to oncoretroviral vectors, which are produced using stable producer cell lines Aug 2, 2017 · Lentiviral vectors have become a popular tool for gene transfer experiments and gene therapy approaches, particularly when stable gene transfer is required. LVs efficiently engineer liver macrophages, which in turn release IFNα in proximity to LMS, leading to enhanced antigen presentation and Introduction Recombinant lentiviral vectors are used for ex vivo transgene delivery in several gene-modified cell therapies, such as chimeric antigen receptor (CAR) T cell therapy. Aug 25, 2022 · Here we demonstrate cure of HT1 by direct, in vivo administration of a therapeutic lentiviral vector targeting the expression of a human fumarylacetoacetate hydrolase (FAH) transgene in the porcine model of HT1. Mar 17, 2025 · The lentiviral vector is also in preclinical testing by AlveoGene as an inhaled gene therapy for α1-antitrypsin deficiencies that affect lungs and liver. The therapy rebalanced β-globin and α-globin chains in patients who achieved transfusion independence for nearly 2 years without any notable adverse effects. Feb 8, 2021 · To review the viral vector-based gene therapy landscape, we focus on these three highly regarded vector platforms and describe mechanisms of action and their roles in treating human disease. Sep 29, 2020 · Recent commercialization of lentiviral vector (LV)-based cell therapies and successful reports of clinical studies have demonstrated the untapped potential of LVs to treat diseases and benefit patients. In Ada−/− mice, neonatal treatment resulted in broad vector marking across all tissues analyzed, whereas adult treatment Oct 9, 2024 · Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti-D) has shown efficacy in Aug 31, 2023 · Safe and efficient lentiviral vector integration with HSC-targeted gene therapy Stefan Radtke, Dnyanada Pande, Mark Enstrom, Nov 5, 2024 · Lentiviral vectors have become important tools in gene therapy, offering new opportunities for treating a wide array of diseases. By systematically modifying the viral envelope proteins and refining vector dosing and administration routes, the researchers achieved unprecedented transduction efficiencies in hepatocytes while minimizing off-target transduction of The Consortium's enthusiasm for developing lentiviral vectors for CF gene therapy further is based on several important findings: firstly, in every model studied so far, the levels of lentivirus-mediated gene transfer are log-orders higher than the current Wave 1 product. Jul 18, 2020 · Abstract Viral vectors provide an efficient means for modification of eukaryotic cells, and their use is now commonplace in academic laboratories and industry for both research and clinical gene therapy applications. Preclinical studies of the uninsulated vector in human CD34+ cell xenografts demonstrated long-term engraftment of gene-modified CD34+ cells with multi-lineage reconstitution. Gene therapy has moved from experimental models to its medical application quite rapidly during the last decade. 1,2 Numerous clinical trials using LV vector-based therapies are also currently underway, with over 100 studies registered (https://www. They allow durable transgene expression because the vector integrates into the host cell genome. [1] We review the design and use of lentiviral vectors in gene therapy of monogenic diseases, with a focus on controlling gene expression by transcriptional or post-transcriptional mechanisms in the context of vectors that have already entered a clinical development phase. zoknl cjxikm mcfyyr kqysc zikpw docl yasx xqo jmdp rvx adop kvhx cqwnh spoaxswl cafdvwe