Despite new medication, cystic fibrosis often leads to permanent lung damage. Working with an international team, researchers ...

Cystic fibrosis (CF) is a single-gene disorder with insufficient treatment options and a target organ, the lung that is relatively easily accessible. Thus, it is not surprising that in the early ...

Using the Human Genome Sequence to Study Disease ... For example, cystic fibrosis is typically characterized as a single-gene disease associated with recessive mutations in the CFTR gene.

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 ...

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 ...

Scientists have created a mega-database revealing how half a million different DNA mutations generate ... such as Huntington's disease and cystic fibrosis. However, in many cases, it's not ...

The Massachusetts-based biotech plans to use the funds to push its candidates into mid-stage clinical trials in a space ...

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 ...

A gene is a fundamental unit of heredity ... This approach has shown promise for treating conditions such as sickle cell anemia, cystic fibrosis, and certain types of cancer. The study of genes and ...

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 ...

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 ...