Kaleido Scope5y
CFTR Rat Models Core
Sharma J, et al: A novel G542X CFTR rat model of cystic fibrosis is sensitive to nonsense mediated decay ... and to support continued studies of cell-based therapies, gene editing techniques, and ...
Medscape11d
Cystic Fibrosis Gene Therapy: Successes, Failures and Hopes for the Future
Viral and nonviral clinical trials have established proof-of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown whether gene transfer efficiency is sufficient ...
Nature1mon
Heterozygote Screening for Cystic Fibrosis
In the sequential model the woman is tested first and ... leading the NIH Workshop on Population Screening for the Cystic Fibrosis Gene to advocate that no mass programmes of heterozygote testing ...
Nature2y
Laboratory standards and guidelines for population-based cystic fibrosis carrier screening
The ACMG charged the Accreditation of Genetic Services Committee, chaired by Dr. Robert Desnick, to establish a Subcommittee on Cystic Fibrosis Carrier ... the screening model to be used (couple ...
Kaleido Scope6y
Functional Assay Core
A number of experimental techniques that involve measurements in engineered CF cell lines (available through the Center’s Gene Expression Core ... and translational science studies in CF in these ...
Science Daily7mon
Cystic Fibrosis News
An international study has identified two types of these so-called dysbioses in cystic fibrosis. They display ... Landmark Study Involving Babies in Ireland Supports Use of Cystic Fibrosis Drug in ...
BBC1y
Gene therapy and cystic fibrosis
An explanation of the cause and effects of the genetic disorder cystic fibrosis and how it can be treated using gene therapy. There is a discussion about gene therapy which includes an interview ...
Business Wire1mon
ReCode Therapeutics Announces Additional Funding From the Cystic Fibrosis Foundation to Accelerate Development of Novel Gene Correction Therapeutics to Treat Cystic Fibrosis
About Cystic Fibrosis Cystic fibrosis (CF) is a progressive genetic disease that causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis ...
New insights into genetic mechanisms could improve treatment of liver fibrosis
The study, "Transcriptomic signatures of progressive and regressive liver fibrosis and portal hypertension,"was published in ...
Hosted on MSN29d
AI Powers Breakthroughs in Gene Therapy
This study opens up new possibilities for treating lung diseases like cystic fibrosis ... the LiON model could also be applied to optimize LNPs for other areas of gene therapy, such as treatments ...
BBC1y
Gene therapy and cystic fibrosis
An explanation of the cause and effects of the genetic disorder cystic fibrosis and how it can be treated using gene therapy. There is a discussion about gene therapy which includes an interview ...