New Combination of CFTR Mutations Found in Chinese Siblings With CF
A combination of two mutations, including one that had never been reported, were found in two Chinese siblings who developed a severe...
Planned CHOICES Trial in Rare CF Mutations Given Regulatory Support
Proteostasis Therapeutics reported receiving scientific advice for its CHOICES clinical trial program, which is to test its personalized...
New CRISPR Tool Fixes CFTR Mutations in CF Patients’ Stem Cells, Study Finds
A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of cystic fibrosis...
Proteostasis On Track to Develop Organoids for Personalized Medicine Strategy for CF Patients with R
A project aimed at personalized medicine based on the use of organoids, or “mini-organs,” from cystic fibrosis (CF) patients with rare...
New Tool for Rapidly Analyzing CRISPR Edits Reveals Frequent Production of Unintended Edits
New method unmasks all DNA changes caused by CRISPR in two days vs two months WILMINGTON, DE — Amidst rising hopes for using CRISPR gene...
Gene Editing Tool Corrects CF-causing Mutations in Patient-derived Cells, Study Shows
Using the CRISPR gene editing tool, a team of researchers was able to permanently correct two mutations in the CFTR gene — mutations that...
FDA Approves Symdeko for Treatment of CF Children, Ages 6 to 11, with Certain CFTR Mutations
The U.S. Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) tablets for the treatment of...
Eluforsen Found to be Safe for Adults with Cystic Fibrosis Caused by F508del Mutation, Study Reports
The experimental treatment eluforsen is safe for adults with cystic fibrosis(CF) caused by a mutation called F508del, a new study...
How to Love Your Body, Even If It’s Genetically Abnormal
When we say “me” or “I,” what exactly are we referring to? Are we referring to our bodies, our minds, or the combination of the two? It’s...
Abeona Therapeutics to Report New Preclinical Data Demonstrating Therapeutic Potential of ABO-401 fo
NEW YORK and CLEVELAND, April 30, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and...
