Last Updated: 09/26/2026
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Found 25 publications
Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease alleles.
Journal: medRxiv : the preprint server for health sciences
Published: April 03, 2026
Monitoring biological effects of somatic cell genome editing.
Journal: Nature reviews. Genetics
Published: October 28, 2025
Generation of WTD, a control human iPSC line for genetic research.
Journal: Stem cell research
Published: August 19, 2025
Haplotype editing with CRISPR-Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: February 10, 2025
Haplotype editing with CRISPR/Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL.
Journal: bioRxiv : the preprint server for biology
Published: January 07, 2025
Generation and characterization of an isogenic control line by correcting the BAG3 P209L mutation of a human induced pluripotent stem cell (hiPSC) line from a patient with myofibrillar myopathy-6.
Journal: Stem cell research
Published: October 22, 2024
Generation and characterization of a human induced pluripotent stem cell (iPSC) line from a patient with BAG3 P209L myofibrillar myopathy-6.
Journal: Stem cell research
Published: October 22, 2024
Characterizing and controlling CRISPR repair outcomes in nondividing human cells.
Journal: Nature communications
Published: September 24, 2024
Neuronal DNA repair reveals strategies to influence CRISPR editing outcomes.
Journal: bioRxiv : the preprint server for biology
Published: July 09, 2024
Advances and challenges in modeling inherited peripheral neuropathies using iPSCs.
Journal: Experimental & molecular medicine
Published: November 10, 2023
Functional analysis of a common BAG3 allele associated with protection from heart failure.
Journal: Nature cardiovascular research
Published: October 17, 2021
Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2E.
Journal: Frontiers in cell and developmental biology
Published: June 09, 2021
Last Updated: 09/26/2026