Gene & Cell Therapy
AAV and lentiviral gene therapy, in vivo CAR-T, iPSC- and stem-cell-derived replacement therapies such as islets and dopaminergic neurons.
Most influential
- Adeno-associated virus vector as a platform for gene therapy deliveryReview2,442 citationsdoi:10.1038/s41573-019-0012-9
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia BArticle1,863 citationsdoi:10.1056/nejmoa1108046
- Adeno-associated virus as a delivery vector for gene therapy of human diseases
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for treating various diseases. However, as rAAV is being more widel…
Abstract excerpt by Jiang-Hui Wang et al., CC-BY, https://doi.org/10.1038/s41392-024-01780-w; truncated.
Review852 citationsdoi:10.1038/s41392-024-01780-w - Adeno-Associated Virus (AAV) as a Vector for Gene TherapyReview1,359 citationsdoi:10.1007/s40259-017-0234-5
- Engineering adeno-associated virus vectors for gene therapyReview1,175 citationsdoi:10.1038/s41576-019-0205-4
- Delivering CRISPR: a review of the challenges and approaches
Gene therapy has long held promise to correct a variety of human diseases and defects. Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the mechanism of the CRISPR-based prokaryotic adaptive immune system (CRISPR-associated system, Cas), and its repurposing into a potent gene editing tool has revolutionized the field of mo…
Abstract excerpt by Christopher A. Lino et al., CC-BY, https://doi.org/10.1080/10717544.2018.1474964; truncated.
Review1,184 citationsdoi:10.1080/10717544.2018.1474964 - Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV VectorsArticle970 citationsdoi:10.1089/hum.2009.182
- Immunogenicity and toxicity of AAV gene therapy
Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in the last decade and has achieved cures of debilitating diseases such as hemophilia A and B. Nevertheless, progress is still being hampered by immune responses against the AAV capsid antigens or the transgene products. Immunosuppression designed to blunt T cell responses …
Abstract excerpt by Hildegund C.J. Ertl, CC-BY, https://doi.org/10.3389/fimmu.2022.975803; truncated.
Review397 citationsdoi:10.3389/fimmu.2022.975803 - Immune responses to AAV vectors: overcoming barriers to successful gene therapyReview955 citationsdoi:10.1182/blood-2013-01-306647
- Functional, metabolic and transcriptional maturation of human pancreatic islets derived from stem cells
Transplantation of pancreatic islet cells derived from human pluripotent stem cells is a promising treatment for diabetes. Despite progress in the generation of stem-cell-derived islets (SC-islets), no detailed characterization of their functional properties has been conducted. Here, we generated functionally mature SC-islets using an optimized protocol and …
Abstract excerpt by Balboa D et al., CC-BY, https://doi.org/10.1038/s41587-022-01219-z; truncated.
Article359 citationsdoi:10.1038/s41587-022-01219-z - Emerging Issues in AAV-Mediated In Vivo Gene TherapyReview879 citationsdoi:10.1016/j.omtm.2017.11.007
- Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challengesReview893 citationsdoi:10.1038/nrg2988
- Current Clinical Applications of In Vivo Gene Therapy with AAVs
Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases affect over 30 million Americans. For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inherited human diseases, offering durable and possibly curative clinical benefit with a single treatme…
Abstract excerpt by Jerry R. Mendell et al., CC-BY, https://doi.org/10.1016/j.ymthe.2020.12.007; truncated.
Review743 citationsdoi:10.1016/j.ymthe.2020.12.007 - Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trialArticle796 citationsdoi:10.1016/s0140-6736(13)62117-0
- Engineering adeno-associated viruses for clinical gene therapyReview761 citationsdoi:10.1038/nrg3742
Newest work
- A Phase Ib/II Open Label Study of Perioperative Navlimetostat Combined With Modified FOLFIRINOX in Patients With MTAP-Deleted Pancreatic CancerClinical trialNCT07905131
- Characterising differential maturation of stem cell-derived islets in mice: Graft function and transcriptomic analysisArticledoi:10.1111/dme.70507
- Report from the 2026 Barth Syndrome Foundation International Scientific, Medical & Family Conference: Advances in Barth Syndrome Research, Therapy Development, and Clinical CareArticledoi:10.3390/jdb14040045
- A Systematic Review of Metachromatic Leukodystrophy Newborn Screening: Transitioning from Pilot Studies to the Real WorldReviewdoi:10.3390/ijns12040086
- Integration-Associated Paraspinal Neoplasm after AAV Gene TherapyArticledoi:10.1056/nejmc2607927
- Cell-autonomous restoration of splicing homeostasis and RP11 phenotype in patient-derived RPE and retinal organoids by PRPF31.AAV gene therapyArticledoi:10.1038/s41467-026-78065-z
- Next-generation CAR-T cell therapy against cancer: precision engineering, programmable immunity, and emerging clinical frontiersReviewdoi:10.1186/s43046-026-00415-1
- Advancing In Vivo Chimeric Antigen Receptor T-Cell Engineering to Accelerate Clinical TranslationReviewdoi:10.1002/mco2.71030
- Efficacy and Safety of GNSC-001 Intra-articular Injection for Knee OsteoarthritisClinical trialNCT07831577
- Intranasal Stem Cells to Treat Perinatal Brain Injury to Combat Cerebral PalsyClinical trialNCT07831746
Clinical trials
- The Cancer of the Pancreas Screening-5 CAPS5)StudyClinical trialNCT02000089
- A Clinical Study of Sacituzumab Tirumotecan (Sac-TMT, MK-2870) in People With Breast Cancer (MK-2870-032)Clinical trialNCT06966700
- ALTTO (Adjuvant Lapatinib And/Or Trastuzumab Treatment Optimisation) Study; BIG 2-06/N063DClinical trialNCT00490139
- Study to Evaluate Efficacy, Immunogenicity and Safety of the Sputnik-LightClinical trialNCT04741061
- Genetic Testing or Clinical Assessment in Determining the Need for Chemotherapy in Women With Breast Cancer That Involves No More Than 3 Lymph NodesClinical trialNCT00433589
- Different Factors Affecting Patients With Newly Diagnosed Bladder CancerClinical trialNCT00553124
- Two Studies for Patients With High Risk Prostate Cancer Testing Less Intense Treatment for Patients With a Low Gene Risk Score and Testing a More Intense Treatment for Patients With a High Gene Risk Score, The PREDICT-RT TrialClinical trialNCT04513717
- Study of Tumor Tissue Testing in Selecting Treatment for Patients With Metastatic or Locally Advanced Colorectal CancerClinical trialNCT00975897