Top Gene Therapy Research Leaders (KOLs) 2021-2026

Top research leaders (by recent publication volume)

#	Researcher	Affiliation	Papers
1	Gao Guangping	University of Massachusetts Chan Medical School	12
2	Xie Jun	University of Massachusetts Chan Medical School	7
3	Qi Jieyu	Zhuhai People's Hospital	6
4	Chai Renjie	Zhongda Hospital	6
5	Mueller Christian	Sanofi	6
6	Miesbach Wolfgang	University Hospital Frankfurt	5
7	Su Qin	Horae Gene Therapy Center	4
8	Wang Dan	University of Massachusetts Chan Medical School	4
9	Yang Yang	West China Hospital	4
10	Li Zhongwan	Exegenesis Bio Inc.	4
11	Chen Shuyuan	Exegenesis Bio Inc.	4
12	Liu Wei	National Clinical Research Centre For Blood Diseases	4
13	Muramatsu Shin-Ichi	Jichi Medical University	4
14	Tan Fangzhi	Zhongda Hospital	4
15	Zhang Liyan	Zhongda Hospital	4
16	Muntoni Francesco	NIHR Great Ormond Street Hospital Biomedical Research Cent	4
17	Elmer Bradford	Sanofi	4
18	Wischhof Erik	Sanofi	4

Ranked by recent publication volume; names not yet disambiguated and not citation-weighted. The full brief adds ORCID disambiguation, citation weighting, and clinical-trial investigator data.

Leading institutions

University of California (12), University of Massachusetts Chan Medical School (11), University of Florida (11), University of Pennsylvania (11), Mayo Clinic (10), College of Pharmacy (10), Inc. (10), West China Hospital (9), College of Veterinary Medicine (8), College of Medicine (8)

Active subtopics

Genetic Therapy (292), Dependovirus (265), Genetic Vectors (248), Mice (159), Disease Models, Animal (109), Gene Therapy Agents (75), Gene Transfer Techniques (58), Mice, Inbred C57BL (48), Gene Editing (40), Transduction, Genetic (34)

Top journals

Molecular therapy : the journal of the American Society of Gene Therapy (54), Molecular therapy. Methods & clinical development (25), Human gene therapy (22), bioRxiv : the preprint server for biology (20), Molecular therapy. Advances (19), International journal of molecular sciences (16)

Representative recent papers

Efficient and Safe Transduction of Cochlear Outer Hair Cells in Adult Mice with AAV2.7m8-Myo15. (2026) · PMID 42478043

Gene/Genome Editing in Cardiovascular Biology and Disease. (2026) · PMID 42474011

AAV Vector-Mediated Modulation of Signaling Pathways in Neurological Disorders: Insights From Cellular, Animal, and Human Studies. (2026) · PMID 42473687

Preclinical study of an optimized AAV cancer vaccine in a spontaneous canine model of oral melanoma. (2026) · PMID 42472000

Restoration, Not Bypass: Otoferlin Gene Therapy and a New Era in Hearing Loss Treatment. (2026) · PMID 42470357

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