For more than a decade a gene therapy involving short strings of bases pairs, known as antisense oligonucleotides (ASOs), has been used to block the production of certain proteins by our body’s ...

Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for ...

Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...

Less than a month after researchers unveiled that a “ticking DNA clock” is behind Huntington’s disease (HD), scientists have now identified an antisense oligonucleotide (ASO) that can slow ...

Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance ...

In 'Bench to Bedside', Joel R. Chamberlain and Jeffrey S. Chamberlain discuss studies using antisense oligonucleotides to treat Duchenne muscular dystrophy and myotonic dystrophy. In 'Bedside to ...

Spinraza treatment does not lead to significant kidney dysfunction in SMA patients with types 1 and 2, a safety and efficacy ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Cure Rare Disease launched its SCA3 program in 2021 to develop an antisense oligonucleotide (ASO) therapy, with initial funding support provided by Gregory Klassen, a patient living with SCA3.

More information: Raul H. Bortolin et al, Antisense Oligonucleotide Therapy for Calmodulinopathy, Circulation (2024). DOI: 10 ...