Nationwide Children's News Releases
Viewing: 11-20 of 96 | All
Jan 20, 2023
New Insights into the Role of the NOTCH1 Gene in Congenital Heart Defects
A new study published in Circulation Research provides novel insights into how mutations in the NOTCH1 gene may lead to abnormal differentiation and proliferation of cardiomyocytes and contribute to ventricular hypoplasia and other structural anomalies found in congenital heart defects in humans.
Aug 18, 2022
New Disorder of Copper Metabolism Identified, Caused by Variants in CTR1 Gene
Copper is essential for many cellular functions, including cellular respiration, antioxidant defense, neurotransmitter biosynthesis and neuropeptide amidation, among others. Until recently, only two inborn errors of copper metabolism were well established. Both are rare. Wilson's disease and Menkes disease result from mutations in the copper-transporting P-type ATPases; ATP7B and ATP7A, respectively.
Mar 09, 2022
Early Phase Gene Therapy Study for SMARD1/CMT2S Disease Now Enrolling
Nationwide Children's Hospital has launched a new Phase I/IIa gene replacement clinical study to evaluate the safety of a gene therapy for IGHMBP2-related diseases, specifically spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S). SMARD1 and CMT2S are rare inherited conditions that are both caused by mutations in the IGHMBP2 gene, of which more than 60 have been found to date. SMARD1 is the most severe form of these diseases and causes muscle weakness and respiratory failure in infancy.
Feb 25, 2022
Dennis Durbin, MD, MSCE, Named President of the Abigail Wexner Research Institute at Nationwide Children’s Hospital
Beginning March 1, 2022, Dennis Durbin, MD, MSCE, will become the third president of the Abigail Wexner Research Institute at Nationwide Children's Hospital. Dr. Durbin joined Nationwide Children's in 2018 as its first chief scientific officer.
Jan 26, 2022
Nationwide Children’s Hospital Researcher Lauren Bakaletz, PhD, Honored by AAAS as Lifetime AAAS Fellow
The American Association for the Advancement of Science (AAAS), the world’s largest general scientific society and publisher of the Science family of journals, has elected Lauren Bakaletz, PhD, director of the Center for Microbial Pathogenesis in the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital to the newest class of AAAS Fellows, among the most distinct honors within the scientific community.
Dec 13, 2021
Human MicroRNA Inhibits Expression of Pathogenic Gene Underlying Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral muscular dystrophy (FSHD) is caused by aberrant expression of the DUX4 gene in skeletal muscles. Researchers at Nationwide Children’s Hospital have recently demonstrated that an endogenous human microRNA, miR-675, inhibits DUX4 expression and protects muscles from DUX4-mediated cell death in a mouse model when administered via gene therapy. They also showed that the small molecule-based treatments that upregulate miR-675 inhibited DUX4 mRNA and DUX4-associated biomarkers in myotubes derived from patients with FSHD.
Mar 18, 2021
Nationwide Children’s Hospital Affiliate Andelyn Biosciences Receives Significant Investment and Enters Into Strategic Partnership with Pall Corporation and Cytiva
(COLUMBUS, Ohio) – Andelyn Biosciences, Inc, a contract development and manufacturing organization (CDMO) and an affiliate company of Nationwide Children’s Hospital, has received a significant, minority investment from Pall Corporation.
Nov 23, 2020
Andelyn Biosciences Breaks Ground for New Commercial-Scale Gene Therapy Manufacturing Facility
Andelyn Biosciences, an affiliate company of Nationwide Children’s Hospital that manufactures gene therapy products for the biotechnology and pharmaceutical industries, has broken ground on its new site located at 1250 Arthur E. Adams Dr. in The Ohio State University’s Innovation District. Opening in 2022, it will be central Ohio’s first commercial-scale Good Manufacturing Practices (cGMP) production facility devoted to gene therapies.
Oct 19, 2020
Jerry R. Mendell, MD, Elected to the Prestigious National Academy of Medicine
(COLUMBUS, Ohio) – Today, Jerry R. Mendell, MD, principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, was elected to the National Academy of Medicine (NAM), the second faculty member from Nationwide
May 01, 2020
Nationwide Children’s Hospital Names CEO of Andelyn Biosciences
Nationwide Children's Hospital is pleased to announce Mayo Pujols as CEO of Andelyn Biosciences, an affiliate company that will manufacture gene therapy products for the biotechnology and pharmaceutical industries.