In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease ... a lung biologist and research director at Cystic Fibrosis Trust, who was not involved in ...

In January 2023, Asklepios BioPharmaceutical entered a research partnership with ReCode to explore a single-vector gene-editing platform. "ReCode to secure funds for cystic fibrosis gene ...

Learn about our Editorial Policies. In a study published in Nature Communications, scientists reported a novel strategy to deliver the CRISPR-Cas9 gene-editing system into the lungs of a cystic ...

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 ...

The versatility empowers ReCode to combine different types of RNA molecules in a single LNP, for example to facilitate gene editing. ReCode’s cystic fibrosis (CF) gene correction proof-of ...

Cystic fibrosis (CF) is a life-threatening genetic disease affecting multiple organ systems, with pancreatic dysfunction representing a critical and often overlooked complication.

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 ...

Gene therapy and gene editing are on the cutting ... company that specializes in developing therapies to treat cystic fibrosis. Vertex also owns the rights to 60% of the profits from sales of ...

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 ...

Researchers at the Broad Institute of MIT and Harvard have developed a gene-editing treatment for prion disease that extends lifespan by about 50% in a mouse model of the fatal neurodegenerative ...