Innovations in gene therapy for the treatment of OTC deficiency

Update on Phase I/II Clinical Trial for Ornithine Transcarbamylase (OTC) Deficiency Using AAV

REW Keynote: Gene Therapy and Gene Editing for Orphan Diseases

Gene Therapy: What Does It Mean for Rare Disease Communities?

ESGCT e-School: Adenoviral vectors – gene delivery tool, powerful weapon in cancer and beyond

Ornithine transcarbamylase (OTC) deficiency with Aimée Dudley, Andrea Gropman, and Tresa Warner

Ornithine Transcarbamylase Deficiency: A Comprehensive Review in Q&A Format

Gene Therapy and Liver Transplants for a Birth Defect

Stanford Course - Principles and Practices of Gene Therapy

VIDEO: OTC medicine helps child with rare genetic condition

The Evolution of Caring for Females With OTC Deficiency

Ultragenyx - Preclinical Evaluation of AAV9-CDKL5 Gene Therapy in Models of CDKL5 Deficiency Disorde

CAR-T Cells: Poseida Therapeutics - Matt Spear

Virology Lectures 2018 #24: Viral Gene Therapy

Global Genes LIVE! Is Gene Therapy Right for Your Disease?

Inherited Disorders of the Urea Cycle

Virology Lectures 2017 #25: Viral Gene Therapy

Rare Disease Day 2019 - Clinical Gene Therapy: The Future is Now

How to Patent Gene Therapies in Europe: Challenges and Strategies - Part 1

How Rare Disease Treatments Get Approved