A preclinical study combining chemically modified suppressor tRNAs with inhaled lipid nanoparticles has restored functional ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...

What you’ll discover in this article ・tRNA-based treatments are a promising approach for genetic diseases caused by nonsense ...

A methylation marker on the tRNA, and a new lipid nanoparticle, helps the ribosome ignore disease-causing mutations ...

The American College of Medical Genetics and Genomics (ACMG) has released updated recommendations for CFTR carrier screening. Pathogenic variants in the CFTR gene can cause cystic fibrosis (CF) as ...

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, ...

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 ...

An experimental drug suggests that a 'path is clearly achievable' to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic ...

Don't bet against Vertex Pharmaceuticals in cystic fibrosis.

Artistic rendering of gene editing reagents — mRNA (red) and DNA (green and yellow) constructs — being packaged into a lipid nanoparticle (blue). UCLA researchers have developed a lipid ...