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 6/30/2021

Scalable mRNA and siRNA Lipid Nanoparticle Production Using a Parallelized Microfluidic Device.

Shepherd SJ, Warzecha CC, Yadavali S, El-Mayta R, Alameh MG, Wang L, Weissman D, Wilson JM, Issadore D, Mitchell MJ

Nano Letters

Publication 5/05/2021

Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia.

Wang L, Muthuramu I, Somanathan S, Zhang H, Bell P, He Z, Yu H, Zhu Y, Tretiakova AP, Wilson JM

Molecular Therapy. Methods & Clinical Development

Publication 2/21/2021

Helper lipid structure influences protein adsorption and delivery of lipid nanoparticles to spleen and liver.

Zhang R, El-Mayta R, Murdoch TJ, Warzecha CC, Billingsley MM, Shepherd SJ, Gong N, Wang L, Wilson JM, Lee D, Mitchell MJ

Biomaterials Science

Publication 2/18/2021

Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9.

Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM

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

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