gene therapy

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

Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus

A Phase I Study of CREPT-618 in Locally Advanced HCC

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

Gene Therapy for IGHMBP2-Related Diseases

A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID

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

ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

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

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

The World Federation of Hemophilia Gene Therapy Registry

Gene Therapy in Treating Patients With Primary Brain Tumors

A Pilot Feasibility Study of Oral 5-Fluorocytosine and Genetically-Modified Neural Stem Cells Expressing E.Coli Cytosine Deaminase for Treatment of Recurrent High Grade Gliomas

Combination Chemotherapy Plus Gene Therapy in Treating Patients With CNS Tumors

Interleukin-2 Gene or Methotrexate in Treating Patients With Recurrent or Refractory Stage III or Stage IV Head and Neck Cancer

Gene Therapy for Gyrate Atrophy

Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia