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Rosalind Franklin University of Medicine and Science


Rosalind Franklin University researchers identify new therapeutic for cystic fibrosis

Researchers at Rosalind Franklin University have identified a new therapeutic approach for treating cystic fibrosis. The treatment uses antisense oligonucleotides to restore CFTR function by removing stop mutations. This strategy has shown promise in treating CF patients with class I mutations and similar types of mutations.

SourceRosalind Franklin University of Medicine and Science·JournalProceedings of the National Academy of Sciences·TypeExperimental study·DateFeb 3, 2022

Rosalind Franklin University Researcher awarded NIH grant for CLN3 Batten disease study

A new therapeutic approach for CLN3 Batten disease has been developed by a team led by Rosalind Franklin University researcher Michelle Hastings. The treatment uses antisense oligonucleotides to target the mutated gene causing the disease, showing promise in rodent models and human cells cultured from patients.

New brain target for potential treatment of social pathology in autism spectrum disorder

A study by Rosalind Franklin University of Medicine and Science identifies a specific brain circuit in the amygdala as crucial for empathy, providing hope for new treatments of social difficulties in autism. The findings suggest that targeting this region may help individuals with autism spectrum disorder develop social abilities.

Study shows potential disease treatment in newborns via drug delivery to amniotic fluid

Researchers at Rosalind Franklin University and Oregon Health & Science University developed a breakthrough study on treating congenital diseases in utero using antisense oligonucleotides injected into the amniotic cavity. The procedure resulted in targeted alteration of gene expression for up to a month after birth.

SourceRosalind Franklin University of Medicine and Science·JournalNucleic Acids Research·DateOct 5, 2016

Scientists prevent development of deafness in animals engineered to have Usher syndrome

Researchers have developed a novel therapeutic approach to treat deafness and balance impairment by injecting mice with an antisense oligonucleotide that targets a specific gene mutation. The treatment, which was tested on mice engineered to model human Usher syndrome, successfully rescues hearing and cures balance problems.