Ami M. Kabadi

Sarepta Therapeutics (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

13h-index3.9kcitations23works0.02yr avg
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Research Topics

CRISPR and Genetic Engineering(16), Pluripotent Stem Cells Research(8), Muscle Physiology and Disorders(5), RNA Interference and Gene Delivery(4), Epigenetics and DNA Methylation(3)

Publications23 total

Characterization of a humanized mouse model of Duchenne muscular dystrophy to support the development of genetic medicines
Disease Models & Mechanisms·2025Open Access
Epigenome editing of the CFTR-locus for treatment of cystic fibrosis
UNC Libraries·2024Open Access
AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities
Current Opinion in Biomedical Engineering·2023· 12 cited
Epigenome editing of the CFTR-locus for treatment of cystic fibrosis
Journal of Cystic Fibrosis·2021· 13 citedOpen Access
Applications of Functional Genomics for Drug Discovery
SLAS DISCOVERY·2020· 13 citedOpen Access
Incomplete MyoD-induced transdifferentiation is associated with chromatin remodeling deficiencies
Nucleic Acids Research·2017· 32 citedOpen Access
Incomplete MyoD-induced transdifferentiation is associated with chromatin remodeling deficiencies
2017· 1 cited
525. Directed Molecular Evolution of Transcription Factors in Mammalian Cells for Enhanced Directed Cell Differentiation
Molecular Therapy·2016Open Access
Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements
Nature Methods·2015· 1.0k citedOpen Access
396. Multiplex CRISPR/Cas9 Genome Engineering for Directing Myogenic Cellular Differentiation
Molecular Therapy·2015Open Access
485. Targeted Epigenome Editing by CRISPR/Cas9-Based Repressors for Silencing of Distal Regulatory Elements
Molecular Therapy·2015· 1 citedOpen Access
Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
Nature Communications·2015· 448 citedOpen Access
Engineering Transcription Factors to Program Cell Fate Decisions
DukeSpace (Duke University)·2015
Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases
Molecular Therapy·2014· 110 citedOpen Access
Enhanced MyoD-Induced Transdifferentiation to a Myogenic Lineage by Fusion to a Potent Transactivation Domain
ACS Synthetic Biology·2014· 45 citedOpen Access
A CRISPR/Cas9-Based System for Reprogramming Cell Lineage Specification
Stem Cell Reports·2014· 200 citedOpen Access
Special Issue on Engineered DNA-Binding Proteins
ACS Synthetic Biology·2014
Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector
Nucleic Acids Research·2014· 380 citedOpen Access
Engineering synthetic TALE and CRISPR/Cas9 transcription factors for regulating gene expression
Methods·2014· 46 citedOpen Access
RNA-guided gene activation by CRISPR-Cas9–based transcription factors
Nature Methods·2013· 1.3k citedOpen Access
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Frequent Co-authors

Charles A. Gersbach(13), Timothy E. Reddy(9), Pratiksha I. Thakore(7), David G. Ousterout(6), Gregory E. Crawford(5), Lingyun Song(4), Pablo Pérez‐Piñera(3), Jennifer B. Kwon(3), Lauren Drowley(3), Christopher M. Vockley(2), Kam W. Leong(2), Farshid Guilak(2), Anthony D’Ippolito(2), Alexias Safi(2), Nishkala K. Shivakumar(2), William H. Majoros(2), Charles A. Gersbach(2), Matthew T. Brown(2), David M. Gooden(2), Dawn M. Z. Schmidt(2)