{"topic":{"slug":"gene-cell-therapy","name":"Gene & Cell Therapy","domain":"medicine","description":"AAV and lentiviral gene therapy, in vivo CAR-T, iPSC- and stem-cell-derived replacement therapies such as islets and dopaminergic neurons.","keywords":["gene therapy","cell therapy"],"paper_count":232,"latest_published":"2026-10-29","url":"https://space-kitty.com/topics/gene-cell-therapy","api_url":"https://space-kitty.com/api/v1/topics/gene-cell-therapy"},"dossier":{"slug":"gene-cell-therapy","topic":"gene-cell-therapy","title":"Gene & Cell Therapy: State of the Field (October 2026)","summary":"Covers adeno-associated virus (AAV) and lentiviral gene therapy, in vivo CAR-T and stem-cell-derived replacement cells such as islets and dopaminergic neurons, with the evidence as of October 2026. The main takeaway: viral gene addition has clinical precedent in several monogenic diseases, but immune responses, dose-linked toxicity and integration questions still bound the field, while in vivo CAR-T and stem-cell replacement remain at early clinical or preclinical stages.","version":1,"published_at":"2026-10-30","updated_at":"2026-10-30","stats":{"words":3785,"citations":57,"claims":34,"timeline":19,"benchmarks":8,"open_problems":6,"key_papers":15,"glossary":16},"verification":{"status":"verified","method":"automated figure and citation-relevance triage against cited abstracts, plus targeted AI review with web checks of flagged statements; automated citation resolution","claims_checked":17,"issues_fixed":16,"verified_at":"2026-10-30"},"ai_assisted":true,"price":{"usd":"0.20","protocol":"x402","network":"eip155:84532","asset":"USDC"},"url":"https://space-kitty.com/dossiers/gene-cell-therapy","preview_url":"https://space-kitty.com/api/v1/dossiers/gene-cell-therapy/preview","purchase_url":"https://space-kitty.com/api/v1/dossiers/gene-cell-therapy"},"top_papers":[{"id":"doi:10.1038/s41573-019-0012-9","kind":"review","title":"Adeno-associated virus vector as a platform for gene therapy delivery","authors":["Dan Wang","Phillip W.L. Tai","Guangping Gao"],"author_count":3,"year":2019,"published":"2019-02-01","venue":"Nature Reviews Drug Discovery","cited_by":2442,"doi":"10.1038/s41573-019-0012-9","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41573-019-0012-9","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41573-019-0012-9"},{"id":"doi:10.1056/nejmoa1108046","kind":"article","title":"Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B","authors":["Amit Chunilal Nathwani","Edward G. D. Tuddenham","Savita Rangarajan","Cecilia Rosales","Jenny McIntosh","David C. Linch"],"author_count":32,"year":2011,"published":"2011-12-10","venue":"New England Journal of Medicine","cited_by":1863,"doi":"10.1056/nejmoa1108046","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1056/nejmoa1108046","archive_url":"https://space-kitty.com/papers/doi:10.1056/nejmoa1108046"},{"id":"doi:10.1038/s41392-024-01780-w","kind":"review","title":"Adeno-associated virus as a delivery vector for gene therapy of human diseases","authors":["Jiang-Hui Wang","Dominic J. Gessler","Wei Zhan","T. F. Gallagher","Guangping Gao"],"author_count":5,"year":2024,"published":"2024-04-02","venue":"Signal Transduction and Targeted Therapy","cited_by":852,"doi":"10.1038/s41392-024-01780-w","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41392-024-01780-w","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41392-024-01780-w"},{"id":"doi:10.1007/s40259-017-0234-5","kind":"review","title":"Adeno-Associated Virus (AAV) as a Vector for Gene Therapy","authors":["Michael F. Naso","Brian E. Tomkowicz","William L. Perry","William R. Strohl"],"author_count":4,"year":2017,"published":"2017-07-01","venue":"BioDrugs","cited_by":1359,"doi":"10.1007/s40259-017-0234-5","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1007/s40259-017-0234-5","archive_url":"https://space-kitty.com/papers/doi:10.1007/s40259-017-0234-5"},{"id":"doi:10.1038/s41576-019-0205-4","kind":"review","title":"Engineering adeno-associated virus vectors for gene therapy","authors":["Chengwen Li","Richard Jude Samulski"],"author_count":2,"year":2020,"published":"2020-02-10","venue":"Nature Reviews Genetics","cited_by":1175,"doi":"10.1038/s41576-019-0205-4","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41576-019-0205-4","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41576-019-0205-4"},{"id":"doi:10.1080/10717544.2018.1474964","kind":"review","title":"Delivering CRISPR: a review of the challenges and approaches","authors":["Christopher A. Lino","Jason C. Harper","James P. Carney","Jerilyn A. Timlin"],"author_count":4,"year":2018,"published":"2018-01-01","venue":"Drug Delivery","cited_by":1184,"doi":"10.1080/10717544.2018.1474964","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1080/10717544.2018.1474964","archive_url":"https://space-kitty.com/papers/doi:10.1080/10717544.2018.1474964"},{"id":"doi:10.1089/hum.2009.182","kind":"article","title":"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","authors":["Sylvie Boutin","Virginie Monteilhet","Philippe Veron","Christian Leborgne","Olivier Benvéniste","M. Montus"],"author_count":7,"year":2010,"published":"2010-01-22","venue":"Human Gene Therapy","cited_by":970,"doi":"10.1089/hum.2009.182","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1089/hum.2009.182","archive_url":"https://space-kitty.com/papers/doi:10.1089/hum.2009.182"},{"id":"doi:10.3389/fimmu.2022.975803","kind":"review","title":"Immunogenicity and toxicity of AAV gene therapy","authors":["Hildegund C.J. Ertl"],"author_count":1,"year":2022,"published":"2022-08-12","venue":"Frontiers in Immunology","cited_by":397,"doi":"10.3389/fimmu.2022.975803","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.3389/fimmu.2022.975803","archive_url":"https://space-kitty.com/papers/doi:10.3389/fimmu.2022.975803"},{"id":"doi:10.1182/blood-2013-01-306647","kind":"review","title":"Immune responses to AAV vectors: overcoming barriers to successful gene therapy","authors":["Federico Mingozzi","Katherine A. High"],"author_count":2,"year":2013,"published":"2013-04-17","venue":"Blood","cited_by":955,"doi":"10.1182/blood-2013-01-306647","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1182/blood-2013-01-306647","archive_url":"https://space-kitty.com/papers/doi:10.1182/blood-2013-01-306647"},{"id":"doi:10.1038/s41587-022-01219-z","kind":"article","title":"Functional, metabolic and transcriptional maturation of human pancreatic islets derived from stem cells","authors":["Balboa D","Barsby T","Lithovius V","Saarimäki-Vire J","Omar-Hmeadi M","Dyachok O"],"author_count":25,"year":2022,"published":"2022-03-03","venue":"Nature biotechnology","cited_by":359,"doi":"10.1038/s41587-022-01219-z","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41587-022-01219-z","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41587-022-01219-z"},{"id":"doi:10.1016/j.omtm.2017.11.007","kind":"review","title":"Emerging Issues in AAV-Mediated In Vivo Gene Therapy","authors":["Pasqualina Colella","Giuseppe Ronzitti","Federico Mingozzi"],"author_count":3,"year":2017,"published":"2017-12-01","venue":"Molecular Therapy — Methods & Clinical Development","cited_by":879,"doi":"10.1016/j.omtm.2017.11.007","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1016/j.omtm.2017.11.007","archive_url":"https://space-kitty.com/papers/doi:10.1016/j.omtm.2017.11.007"},{"id":"doi:10.1038/nrg2988","kind":"review","title":"Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges","authors":["Federico Mingozzi","Katherine A. High"],"author_count":2,"year":2011,"published":"2011-04-18","venue":"Nature Reviews Genetics","cited_by":893,"doi":"10.1038/nrg2988","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/nrg2988","archive_url":"https://space-kitty.com/papers/doi:10.1038/nrg2988"},{"id":"doi:10.1016/j.ymthe.2020.12.007","kind":"review","title":"Current Clinical Applications of In Vivo Gene Therapy with AAVs","authors":["Jerry R. Mendell","Samiah A. Al-Zaidy","Louise R. Rodino‐Klapac","Kimberly Goodspeed","Steven James Gray","Christine Nichols Kay"],"author_count":13,"year":2020,"published":"2020-12-10","venue":"Molecular Therapy","cited_by":743,"doi":"10.1016/j.ymthe.2020.12.007","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1016/j.ymthe.2020.12.007","archive_url":"https://space-kitty.com/papers/doi:10.1016/j.ymthe.2020.12.007"},{"id":"doi:10.1016/s0140-6736(13)62117-0","kind":"article","title":"Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial","authors":["Robert Edward MacLaren","Markus Groppe","Alun R. Barnard","Charles L. Cottriall","Tanya Tolmachova","Leonard William Seymour"],"author_count":14,"year":2014,"published":"2014-01-15","venue":"The Lancet","cited_by":796,"doi":"10.1016/s0140-6736(13)62117-0","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1016/s0140-6736(13)62117-0","archive_url":"https://space-kitty.com/papers/doi:10.1016/s0140-6736(13)62117-0"},{"id":"doi:10.1038/nrg3742","kind":"review","title":"Engineering adeno-associated viruses for clinical gene therapy","authors":["Melissa A. Kotterman","David V. Schaffer"],"author_count":2,"year":2014,"published":"2014-05-20","venue":"Nature Reviews Genetics","cited_by":761,"doi":"10.1038/nrg3742","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/nrg3742","archive_url":"https://space-kitty.com/papers/doi:10.1038/nrg3742"}],"newest_papers":[{"id":"nct:NCT07905131","kind":"trial","title":"A Phase Ib/II Open Label Study of Perioperative Navlimetostat Combined With Modified FOLFIRINOX in Patients With MTAP-Deleted Pancreatic Cancer","authors":["Daniel Nussbaum, M.D."],"author_count":1,"year":2026,"published":"2026-10-29","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT07905131","source_url":"https://clinicaltrials.gov/study/NCT07905131","archive_url":"https://space-kitty.com/papers/nct:NCT07905131"},{"id":"doi:10.1111/dme.70507","kind":"article","title":"Characterising differential maturation of stem cell-derived islets in mice: Graft function and transcriptomic analysis","authors":["Sullivan R","Gatward LFD","Hong TW","Lyu Z","Whines WTB","Duff C"],"author_count":13,"year":2026,"published":"2026-10-25","venue":"Diabetic medicine : a journal of the British Diabetic Association","cited_by":0,"doi":"10.1111/dme.70507","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1111/dme.70507","archive_url":"https://space-kitty.com/papers/doi:10.1111/dme.70507"},{"id":"doi:10.3390/jdb14040045","kind":"article","title":"Report from the 2026 Barth Syndrome Foundation International Scientific, Medical & Family Conference: Advances in Barth Syndrome Research, Therapy Development, and Clinical Care","authors":["Marjoram L"],"author_count":1,"year":2026,"published":"2026-10-19","venue":"Journal of developmental biology","cited_by":0,"doi":"10.3390/jdb14040045","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.3390/jdb14040045","archive_url":"https://space-kitty.com/papers/doi:10.3390/jdb14040045"},{"id":"doi:10.3390/ijns12040086","kind":"review","title":"A Systematic Review of Metachromatic Leukodystrophy Newborn Screening: Transitioning from Pilot Studies to the Real World","authors":["Waser S","Kistler S","Adang LA","Orchard PJ","Datta AN","Sondheimer N"],"author_count":8,"year":2026,"published":"2026-10-17","venue":"International journal of neonatal screening","cited_by":0,"doi":"10.3390/ijns12040086","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.3390/ijns12040086","archive_url":"https://space-kitty.com/papers/doi:10.3390/ijns12040086"},{"id":"doi:10.1056/nejmc2607927","kind":"article","title":"Integration-Associated Paraspinal Neoplasm after AAV Gene Therapy","authors":["Vo JN","Wu YM","Heider A","Cao X","Wang R","Pham T"],"author_count":12,"year":2026,"published":"2026-10-01","venue":"The New England journal of medicine","cited_by":0,"doi":"10.1056/nejmc2607927","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1056/nejmc2607927","archive_url":"https://space-kitty.com/papers/doi:10.1056/nejmc2607927"},{"id":"doi:10.1038/s41467-026-78065-z","kind":"article","title":"Cell-autonomous restoration of splicing homeostasis and RP11 phenotype in patient-derived RPE and retinal organoids by PRPF31.AAV gene therapy","authors":["Elia M","Pauzuolyte V","Georgiou M","Basche M","Hansohn C","Vasconcelos EJR"],"author_count":15,"year":2026,"published":"2026-09-30","venue":"Nature communications","cited_by":0,"doi":"10.1038/s41467-026-78065-z","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41467-026-78065-z","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41467-026-78065-z"},{"id":"doi:10.1186/s43046-026-00415-1","kind":"review","title":"Next-generation CAR-T cell therapy against cancer: precision engineering, programmable immunity, and emerging clinical frontiers","authors":["Mustaffa FE","Aslam MS","Junaid M"],"author_count":3,"year":2026,"published":"2026-09-29","venue":"Journal of the Egyptian National Cancer Institute","cited_by":0,"doi":"10.1186/s43046-026-00415-1","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1186/s43046-026-00415-1","archive_url":"https://space-kitty.com/papers/doi:10.1186/s43046-026-00415-1"},{"id":"doi:10.1002/mco2.71030","kind":"review","title":"Advancing In Vivo Chimeric Antigen Receptor T-Cell Engineering to Accelerate Clinical Translation","authors":["Bai Z","Yi M","Hu S","Xue D","Wei L","Zhang L"],"author_count":7,"year":2026,"published":"2026-09-27","venue":"MedComm","cited_by":0,"doi":"10.1002/mco2.71030","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1002/mco2.71030","archive_url":"https://space-kitty.com/papers/doi:10.1002/mco2.71030"},{"id":"nct:NCT07831577","kind":"trial","title":"Efficacy and Safety of GNSC-001 Intra-articular Injection for Knee Osteoarthritis","authors":["Genascence Corporation"],"author_count":1,"year":2026,"published":"2026-09-21","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT07831577","source_url":"https://clinicaltrials.gov/study/NCT07831577","archive_url":"https://space-kitty.com/papers/nct:NCT07831577"},{"id":"nct:NCT07831746","kind":"trial","title":"Intranasal Stem Cells to Treat Perinatal Brain Injury to Combat Cerebral Palsy","authors":["UMC Utrecht"],"author_count":1,"year":2026,"published":"2026-09-21","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT07831746","source_url":"https://clinicaltrials.gov/study/NCT07831746","archive_url":"https://space-kitty.com/papers/nct:NCT07831746"}],"related_topics":[{"slug":"cancer-immunotherapy","name":"Cancer Immunotherapy","api_url":"https://space-kitty.com/api/v1/topics/cancer-immunotherapy"},{"slug":"mrna-cancer-vaccines","name":"mRNA Therapeutics & Cancer Vaccines","api_url":"https://space-kitty.com/api/v1/topics/mrna-cancer-vaccines"},{"slug":"gene-editing-therapies","name":"CRISPR & Gene-Editing Therapies","api_url":"https://space-kitty.com/api/v1/topics/gene-editing-therapies"},{"slug":"glp1-metabolic","name":"GLP-1 & Incretin Therapies","api_url":"https://space-kitty.com/api/v1/topics/glp1-metabolic"},{"slug":"neurodegeneration","name":"Alzheimer's, Parkinson's & Neurodegeneration","api_url":"https://space-kitty.com/api/v1/topics/neurodegeneration"},{"slug":"aging-longevity","name":"Biology of Aging & Longevity","api_url":"https://space-kitty.com/api/v1/topics/aging-longevity"},{"slug":"infectious-disease-amr","name":"Infectious Disease, Vaccines & Antimicrobial Resistance","api_url":"https://space-kitty.com/api/v1/topics/infectious-disease-amr"},{"slug":"ai-medicine","name":"AI in Clinical Medicine","api_url":"https://space-kitty.com/api/v1/topics/ai-medicine"},{"slug":"cardiovascular-innovation","name":"Cardiovascular Therapeutics","api_url":"https://space-kitty.com/api/v1/topics/cardiovascular-innovation"}],"premium":{"dossier":{"url":"https://space-kitty.com/api/v1/dossiers/gene-cell-therapy","price_usd":"0.20"},"research_pack":{"url":"https://space-kitty.com/api/v1/topics/gene-cell-therapy/pack","price_usd":"0.20"}},"more":"https://space-kitty.com/api/v1/topics/gene-cell-therapy/papers?sort=top&limit=100"}