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174,212 results for “Powell Gene Therapy Center”

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A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease

31 participants · First posted Jun 10, 2025

A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy

1 participants · First posted May 21, 2025

Not yet recruiting

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

7 participants · First posted Jun 5, 2020

A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC

10 participants · First posted Jul 15, 2011

Gene Discovery Core, The Manton Center

10,000 participants · First posted Apr 19, 2016

Gene Therapy for X Linked Severe Combined Immunodeficiency

10 participants · First posted Feb 27, 2020

An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3

First posted Apr 18, 2023

Gene Therapy for IGHMBP2-Related Diseases

10 participants · First posted Dec 10, 2021

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

10 participants · First posted Sep 9, 2014

Enrolling By Invitation

Gene Therapy Follow up Protocol for Subjects Previously Enrolled in NCI Center for Immuno-Oncology Studies

1,000 participants · First posted Feb 12, 2020

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