Krystal Biotech Touts Positive KB407 CF Gene Therapy Data, Eyes Repeat Dosing and Registrational Path

Krystal Biotech (NASDAQ:KRYS) said it has generated positive interim clinical results for its inhaled cystic fibrosis (CF) ...
Nature

CFTR Gene Mutations in Adults with Disseminated Bronchiectasis

The severity and type of clinical manifestations are variable in patients with cystic fibrosis (CF). The respiratory syndromes in these patients consist of lung infections associated with disseminated ...
News Medical

CFTR modulator can be safe, well-tolerated for younger children with common form of cystic fibrosis

Children ages two to five who have the most common form of cystic fibrosis (CF), caused by two copies of the F508 gene mutation, have not had any modulator treatments available to them until recently.
Nature

Use of MALDI-TOF mass spectrometry in a 51-mutation test for cystic fibrosis: Evidence that 3199del6 is a disease-causing mutation

Purpose: We developed a 51-mutation extended cystic fibrosis (CF) panel that incorporates the 25 previously recommended CFTR mutations, plus 26 additional mutations including 3199del6, which was ...
Healthline

Can Gene Therapy Help Cystic Fibrosis?

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...
EurekAlert!

Modifier gene may explain why some with cystic fibrosis are less prone to infection

Cystic fibrosis is caused by an inherited mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Due to this mutation, the CFTR protein doesn't embed in cell membranes to ...
Kaleido Scope

Animal and Preclinical Models Core

Established in 2002, the Animal and Preclinical Models Core serves to provide specialized capabilities for developing and characterizing numerous CF animal models relevant to CF pathogenesis and ...
Healio

Aerosol gene therapy safe, heightens CFTR protein levels in cystic fibrosis

Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...
Healthline

Can Genetic Testing Predict Cystic Fibrosis?

Genetic testing plays an important role in cystic fibrosis screening and diagnosis. It can help determine if a person has cystic fibrosis or carries gene mutations that cause it. Other tests can also ...
News Medical

ACMG: Technical standard on CFTR variant testing updated

It has been nearly two decades since the American College of Medical Genetics and Genomics (ACMG), together with the American College of Obstetricians and Gynecologists, made the landmark ...
Healio

Q&A: Genetic technologies aim to advance cystic fibrosis treatments

Please provide your email address to receive an email when new articles are posted on . The Cystic Fibrosis Foundation is providing funds to Prime Medicine to advance research on its gene editing ...
JSTOR Daily

A Shortened Adeno-Associated virus Expression Cassette for CFTR Gene Transfer to Cystic Fibrosis Airway Epithelia

Adeno-associated viruses (AAVs) such as AAV5 that transduce airway epithelia from the apical surface are attractive vectors for gene transfer in cystic fibrosis (CF). However, their utility in CF has ...