Area of research
Genetics · Molecular Biology
Research interest
Research focused on Genetic enhancement and Capsid, with related work in Factor IX, Computational biology, Viral vector. Notable publications include 'Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants', 'Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation', and 'Liver-directed lentiviral gene therapy in a dog model of hemophilia B'.
Hemophilia Gene Therapy: The End of the Beginning?
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders
Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy
First hemophilia B gene therapy approved: More than two decades in the making
Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants
Next-generation muscle-directed gene therapy by in silico vector design
Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells
Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9
Preclinical and clinical advances in transposon-based gene therapy
Transposons: Moving Forward from Preclinical Studies to Clinical Trials
A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice
Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD/SCIDγc‐/‐ mice
Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation
Liver-directed lentiviral gene therapy in a dog model of hemophilia B
Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells
Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy
Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver
Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates
Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy
Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy
Low hippocampal PI(4,5)P2 contributes to reduced cognition in old mice as a result of loss of MARCKS
Gene therapy for hemophilia
Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases
Recent developments in transposon-mediated gene therapy
Repression of Cardiac Hypertrophy by KLF15: Underlying Mechanisms and Therapeutic Implications