We invite you to discover Human Gene Therapy Clinical Development, a peer-reviewed journal publishing preclinical research and clinical trial data for gene and cell therapies.

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Publication 6/30/2020

Moving forward after two deaths in a gene therapy trial of myotubular myopathy.

Wilson JM, Flotte TR

Human Gene Therapy

Publication 4/18/2020

Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration.

Hinderer C, Katz N, Dyer C, Goode T, Johansson J, Bell P, Richman L, Buza E, Wilson JM

Molecular Therapy. Methods & Clinical Development

Publication 3/17/2020

ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing.

Breton C, Clark PM, Wang L, Greig JA, Wilson JM

Bmc Genomics

Publication 2/12/2020

A Mutation-Independent CRISPR-Cas9-Mediated Gene Targeting Approach to Treat a Murine Model of Ornithine Transcarbamylase Deficiency

Wang L, Yang Y, Breton C, Bell P, Li M, Zhang J, Che Y, Saveliev A, He Z, White J, Latshaw C, Xu C, McMenamin D, Yu H, Morizono H, Batshaw ML, Wilson JM

Science Advances

Publication 1/24/2020

Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial.

Somanathan S, Calcedo R, Wilson JM

Molecular Therapy : The Journal Of The American Society Of Gene Therapy

Publication 12/28/2019

Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening.

Guimaraes PPG, Zhang R, Spektor R, Tan M, Chung A, Billingsley MM, El-Mayta R, Riley RS, Wang L, Wilson JM, Mitchell MJ

Journal Of Controlled Release : Official Journal Of The Controlled Release Society