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 5/08/2019

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier.

Hordeaux J, Yuan Y, Clark PM, Wang Q, Martino RA, Sims JJ, Bell P, Raymond A, Stanford WL, Wilson JM

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

Publication 4/24/2019

Safe and sustained expression of human iduronidase after adeno-associated virus 9 intrathecal administration in infant rhesus monkeys.

Hordeaux J, Hinderer C, Buza EL, Louboutin JP, Jahan T, Bell P, Chichester JA, Tarantal AF, Wilson JM

Human Gene Therapy

Publication 4/11/2019

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX knockout mice.

Wang L, Yang Y, Breton CA, White J, Zhang J, Che Y, Saveliev A, McMenamin D, He Z, Latshaw C, Li M, Wilson JM

Blood

Publication 3/01/2019

A Birds-Eye View: An Interview with Nick Leschly.

Wilson JM

Human Gene Therapy. Clinical Development

Publication 3/01/2019

A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.

Greig JA, Nordin JML, Smith MK, Ashley SN, Draper C, Zhu Y, Bell P, Buza EL, Wilson JM

Human Gene Therapy. Clinical Development

Publication 12/05/2018

Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function.

Giles AR, Sims JJ, Turner KB, Govindasamy L, Alvira MR, Lock M, Wilson JM

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