BioCentury | May 5, 2020
Deals

BioMarin builds on gene therapy momentum with DiNAQOR deal in cardiomyopathy

...programs. Financial terms were not disclosed. DiNAQOR’s lead program, DiNA-001, is a gene therapy for MYBPC3-linked...
...likely to benefit from the gene therapy in terms of disease stage. Targets MYBPC3 - Myosin binding protein C cardiac Lauren...
BioCentury | Jun 4, 2019
Tools & Techniques

OHSU's Mitalipov proposes path forward for germline gene editing

...by NIH. In a 2017 Nature study they detailed use of CRISPR-Cas9 to correct the MYBPC3...
...Targets: Cas9 - CRISPR-associated protein 9; CCR5 (CD195) - CC chemokine receptor 5; MYBPC3 - myosin binding protein C cardiac...
...University Salk Institute for Biological Studies University of California Berkeley CC chemokine receptor 5 (CCR5) (CD195) CRISPR-associated protein 9 (Cas9) Myosin binding protein C cardiac (MYBPC3) RAD51...
BioCentury | Dec 1, 2018
Politics, Policy & Law

China’s germline growing pain

...Health & Science University (OHSU) used CRISPR-Cas9 editing on 58 human embryos to correct the myosin binding protein C cardiac (MYBPC3)...
BioCentury | Nov 17, 2018
Product Development

MyoKardia’s myosin mechanism deconvolution

...the “important players” the company has studied include the scaffold proteins titin (TTN) and actin; myosin binding protein C cardiac (MYBPC3)...
BioCentury | Aug 22, 2018
Translation in Brief

Confirming CRISPR

...Science University researchers suggested they used CRISPR-Cas 9 editing to correct a mutation in the myosin binding protein C cardiac (MYBPC3)...
...healthy donor eggs from 12 women and sperm from one hypertrophic cardiomyopathy patient carrying the MYBPC3...
...did not perform experiments to exclude this possibility. “The existence of large deletions at the MYBPC3...
BioCentury | Sep 14, 2017
Translation in Brief

New Therapeutic Targets and Biomarkers: August 2017

...University of California San Diego ; University of Cincinnati College of Medicine Distillery Therapeutics Cardiomyopathy Myosin binding protein C cardiac (MYBPC3)...
...CRISPR-associated protein 9) gene editing could be used to correct hypertrophic cardiomyopathy-causing mutations in the MYBPC3...
BioCentury | Aug 9, 2017
Distillery Techniques

Drug platforms

...women and sperm from a patient with hypertrophic cardiomyopathy harboring a heterozygous mutation in the myosin binding protein C cardiac (MYBPC3)...
...gene. In 58 oocytes, microinjection of recombinant Cas9 protein, a single-guide RNA (sgRNA) targeting the MYBPC3...
...Health & Science University, Portland, Ore. email: mitalipo@ohsu.edu Lauren Martz Oregon Health & Science University CRISPR-associated protein 9 (Cas9) Myosin binding protein C cardiac (MYBPC3)...
BioCentury | Aug 3, 2017
Tools & Techniques

The other side of the Rubicon

...Nature , involved CRISPR- Cas9 editing on 58 human embryos carrying a mutation in the MYBPC3...
...will be untreatable diseases where pre-implantation genetic diagnosis (PGD) is not an option. However, the MYBPC3...
...onset BRCA2 - Breast cancer 2 early onset Cas9 - CRISPR-associated protein 9 MYBPC3 - Myosin binding protein C cardiac...
BioCentury | Aug 2, 2017
Preclinical News

U.S. researchers buck embryo editing guidelines

...and should prompt the Academies to revisit their position. The group edited a mutation in myosin binding protein C cardiac (MYBPC3)...
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