# Gene & Cell Therapy Domain: Advanced Medicine · 232 papers indexed · newest 2026-10-29 AAV and lentiviral gene therapy, in vivo CAR-T, iPSC- and stem-cell-derived replacement therapies such as islets and dopaminergic neurons. Key concepts: gene therapy, cell therapy ## Most influential - **[Adeno-associated virus vector as a platform for gene therapy delivery](https://space-kitty.com/papers/doi:10.1038/s41573-019-0012-9)**. Dan Wang, Phillip W.L. Tai, Guangping Gao · 2019 · Nature Reviews Drug Discovery (doi:10.1038/s41573-019-0012-9 · 2,442 citations). Source: - **[Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B](https://space-kitty.com/papers/doi:10.1056/nejmoa1108046)**. Amit Chunilal Nathwani, Edward G. D. Tuddenham, Savita Rangarajan et al. · 2011 · New England Journal of Medicine (doi:10.1056/nejmoa1108046 · 1,863 citations). Source: - **[Adeno-associated virus as a delivery vector for gene therapy of human diseases](https://space-kitty.com/papers/doi:10.1038/s41392-024-01780-w)**. Jiang-Hui Wang, Dominic J. Gessler, Wei Zhan et al. · 2024 · Signal Transduction and Targeted Therapy (doi:10.1038/s41392-024-01780-w · 852 citations). Source: > 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 widely used as a therapy, the increased demand has created challenges for the existing manufacturing methods. Seven rAAV-based gene therapy products have received regulatory approval, but there continue to be concerns about safely using high-dos… > _Abstract excerpt by Jiang-Hui Wang et al., CC-BY, https://doi.org/10.1038/s41392-024-01780-w; truncated._ - **[Adeno-Associated Virus (AAV) as a Vector for Gene Therapy](https://space-kitty.com/papers/doi:10.1007/s40259-017-0234-5)**. Michael F. Naso, Brian E. Tomkowicz, William L. Perry et al. · 2017 · BioDrugs (doi:10.1007/s40259-017-0234-5 · 1,359 citations). Source: - **[Engineering adeno-associated virus vectors for gene therapy](https://space-kitty.com/papers/doi:10.1038/s41576-019-0205-4)**. Chengwen Li, Richard Jude Samulski · 2020 · Nature Reviews Genetics (doi:10.1038/s41576-019-0205-4 · 1,175 citations). Source: - **[Delivering CRISPR: a review of the challenges and approaches](https://space-kitty.com/papers/doi:10.1080/10717544.2018.1474964)**. Christopher A. Lino, Jason C. Harper, James P. Carney et al. · 2018 · Drug Delivery (doi:10.1080/10717544.2018.1474964 · 1,184 citations). Source: > 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 molecular biology and generated excitement for new and improved gene therapies. Additionally, the simplicity and flexibility of the CRISPR/Cas9 site-specific nuclease system has led to its widespread use in many biological research areas incl… > _Abstract excerpt by Christopher A. Lino et al., CC-BY, https://doi.org/10.1080/10717544.2018.1474964; truncated._ - **[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 Vectors](https://space-kitty.com/papers/doi:10.1089/hum.2009.182)**. Sylvie Boutin, Virginie Monteilhet, Philippe Veron et al. · 2010 · Human Gene Therapy (doi:10.1089/hum.2009.182 · 970 citations). Source: - **[Immunogenicity and toxicity of AAV gene therapy](https://space-kitty.com/papers/doi:10.3389/fimmu.2022.975803)**. Hildegund C.J. Ertl · 2022 · Frontiers in Immunology (doi:10.3389/fimmu.2022.975803 · 397 citations). Source: > 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 has shown success in some patients but failed in others especially if they received very high AAV vectors doses. Although it was initially thought that AAV vectors induce only marginal innate responses below the threshold of systemic sympto… > _Abstract excerpt by Hildegund C.J. Ertl, CC-BY, https://doi.org/10.3389/fimmu.2022.975803; truncated._ - **[Immune responses to AAV vectors: overcoming barriers to successful gene therapy](https://space-kitty.com/papers/doi:10.1182/blood-2013-01-306647)**. Federico Mingozzi, Katherine A. High · 2013 · Blood (doi:10.1182/blood-2013-01-306647 · 955 citations). Source: - **[Functional, metabolic and transcriptional maturation of human pancreatic islets derived from stem cells](https://space-kitty.com/papers/doi:10.1038/s41587-022-01219-z)**. Balboa D, Barsby T, Lithovius V et al. · 2022 · Nature biotechnology (doi:10.1038/s41587-022-01219-z · 359 citations). Source: > 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 benchmarked them comprehensively against primary adult islets. Biphasic glucose-stimulated insulin secretion developed during in vitro maturation, associated with cytoarchitectural reorganization and the increasing presence of alpha cells. … > _Abstract excerpt by Balboa D et al., CC-BY, https://doi.org/10.1038/s41587-022-01219-z; truncated._ - **[Emerging Issues in AAV-Mediated In Vivo Gene Therapy](https://space-kitty.com/papers/doi:10.1016/j.omtm.2017.11.007)**. Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi · 2017 · Molecular Therapy — Methods & Clinical Development (doi:10.1016/j.omtm.2017.11.007 · 879 citations). Source: - **[Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges](https://space-kitty.com/papers/doi:10.1038/nrg2988)**. Federico Mingozzi, Katherine A. High · 2011 · Nature Reviews Genetics (doi:10.1038/nrg2988 · 893 citations). Source: - **[Current Clinical Applications of In Vivo Gene Therapy with AAVs](https://space-kitty.com/papers/doi:10.1016/j.ymthe.2020.12.007)**. Jerry R. Mendell, Samiah A. Al-Zaidy, Louise R. Rodino‐Klapac et al. · 2020 · Molecular Therapy (doi:10.1016/j.ymthe.2020.12.007 · 743 citations). Source: > 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 treatment. This review is limited to gene therapy using adeno-associated virus (AAV) because the gene delivered by this vector does not integrate into the patient genome and has a low immunogenicity. There are now five treatments approved for comm… > _Abstract excerpt by Jerry R. Mendell et al., CC-BY, https://doi.org/10.1016/j.ymthe.2020.12.007; truncated._ - **[Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial](https://space-kitty.com/papers/doi:10.1016/s0140-6736(13)62117-0)**. Robert Edward MacLaren, Markus Groppe, Alun R. Barnard et al. · 2014 · The Lancet (doi:10.1016/s0140-6736(13)62117-0 · 796 citations). Source: - **[Engineering adeno-associated viruses for clinical gene therapy](https://space-kitty.com/papers/doi:10.1038/nrg3742)**. Melissa A. Kotterman, David V. Schaffer · 2014 · Nature Reviews Genetics (doi:10.1038/nrg3742 · 761 citations). Source: ## Newest - **[A Phase Ib/II Open Label Study of Perioperative Navlimetostat Combined With Modified FOLFIRINOX in Patients With MTAP-Deleted Pancreatic Cancer](https://space-kitty.com/papers/nct:NCT07905131)**. Daniel Nussbaum, M.D. · 2026 · ClinicalTrials.gov (NCT07905131). Source: - **[Characterising differential maturation of stem cell-derived islets in mice: Graft function and transcriptomic analysis](https://space-kitty.com/papers/doi:10.1111/dme.70507)**. Sullivan R, Gatward LFD, Hong TW et al. · 2026 · Diabetic medicine : a journal of the British Diabetic Association (doi:10.1111/dme.70507). Source: - **[Report from the 2026 Barth Syndrome Foundation International Scientific, Medical & Family Conference: Advances in Barth Syndrome Research, Therapy Development, and Clinical Care](https://space-kitty.com/papers/doi:10.3390/jdb14040045)**. Marjoram L · 2026 · Journal of developmental biology (doi:10.3390/jdb14040045). Source: - **[A Systematic Review of Metachromatic Leukodystrophy Newborn Screening: Transitioning from Pilot Studies to the Real World](https://space-kitty.com/papers/doi:10.3390/ijns12040086)**. Waser S, Kistler S, Adang LA et al. · 2026 · International journal of neonatal screening (doi:10.3390/ijns12040086). Source: - **[Integration-Associated Paraspinal Neoplasm after AAV Gene Therapy](https://space-kitty.com/papers/doi:10.1056/nejmc2607927)**. Vo JN, Wu YM, Heider A et al. · 2026 · The New England journal of medicine (doi:10.1056/nejmc2607927). Source: - **[Cell-autonomous restoration of splicing homeostasis and RP11 phenotype in patient-derived RPE and retinal organoids by PRPF31.AAV gene therapy](https://space-kitty.com/papers/doi:10.1038/s41467-026-78065-z)**. Elia M, Pauzuolyte V, Georgiou M et al. · 2026 · Nature communications (doi:10.1038/s41467-026-78065-z). Source: - **[Next-generation CAR-T cell therapy against cancer: precision engineering, programmable immunity, and emerging clinical frontiers](https://space-kitty.com/papers/doi:10.1186/s43046-026-00415-1)**. Mustaffa FE, Aslam MS, Junaid M · 2026 · Journal of the Egyptian National Cancer Institute (doi:10.1186/s43046-026-00415-1). Source: - **[Advancing In Vivo Chimeric Antigen Receptor T-Cell Engineering to Accelerate Clinical Translation](https://space-kitty.com/papers/doi:10.1002/mco2.71030)**. Bai Z, Yi M, Hu S et al. · 2026 · MedComm (doi:10.1002/mco2.71030). Source: - **[Efficacy and Safety of GNSC-001 Intra-articular Injection for Knee Osteoarthritis](https://space-kitty.com/papers/nct:NCT07831577)**. Genascence Corporation · 2026 · ClinicalTrials.gov (NCT07831577). Source: - **[Intranasal Stem Cells to Treat Perinatal Brain Injury to Combat Cerebral Palsy](https://space-kitty.com/papers/nct:NCT07831746)**. UMC Utrecht · 2026 · ClinicalTrials.gov (NCT07831746). Source: ## Clinical trials - **[The Cancer of the Pancreas Screening-5 CAPS5)Study](https://space-kitty.com/papers/nct:NCT02000089)**. Johns Hopkins University · 2013 · ClinicalTrials.gov (NCT02000089). Source: - **[A Clinical Study of Sacituzumab Tirumotecan (Sac-TMT, MK-2870) in People With Breast Cancer (MK-2870-032)](https://space-kitty.com/papers/nct:NCT06966700)**. Merck Sharp & Dohme LLC · 2025 · ClinicalTrials.gov (NCT06966700). Source: - **[ALTTO (Adjuvant Lapatinib And/Or Trastuzumab Treatment Optimisation) Study; BIG 2-06/N063D](https://space-kitty.com/papers/nct:NCT00490139)**. Novartis Pharmaceuticals · 2007 · ClinicalTrials.gov (NCT00490139). Source: - **[Study to Evaluate Efficacy, Immunogenicity and Safety of the Sputnik-Light](https://space-kitty.com/papers/nct:NCT04741061)**. Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation · 2021 · ClinicalTrials.gov (NCT04741061). Source: - **[Genetic Testing or Clinical Assessment in Determining the Need for Chemotherapy in Women With Breast Cancer That Involves No More Than 3 Lymph Nodes](https://space-kitty.com/papers/nct:NCT00433589)**. European Organisation for Research and Treatment of Cancer - EORTC · 2007 · ClinicalTrials.gov (NCT00433589). Source: - **[Different Factors Affecting Patients With Newly Diagnosed Bladder Cancer](https://space-kitty.com/papers/nct:NCT00553124)**. University Hospital Birmingham · 2007 · ClinicalTrials.gov (NCT00553124). Source: - **[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 Trial](https://space-kitty.com/papers/nct:NCT04513717)**. NRG Oncology · 2020 · ClinicalTrials.gov (NCT04513717). Source: - **[Study of Tumor Tissue Testing in Selecting Treatment for Patients With Metastatic or Locally Advanced Colorectal Cancer](https://space-kitty.com/papers/nct:NCT00975897)**. Medical Research Council · 2009 · ClinicalTrials.gov (NCT00975897). Source: --- Space Kitty Archive · https://space-kitty.com · API: https://space-kitty.com/api/v1 · MCP: https://space-kitty.com/mcp · Premium: $0.20/request via x402 (Base Sepolia (testnet)). Metadata: arXiv (CC0), OpenAlex (CC0), Europe PMC (CC BY/CC0 works), ClinicalTrials.gov.