In a new study, Chinese researchers have discovered the previously unrecognized role of alternative splicing of the DOC2A ...

Alternative splicing is a genetic process where different segments of genes are removed, and the remaining pieces are joined together during transcription to messenger RNA (mRNA). This mechanism ...

Scientists at the Hospital for Sick Children (SickKids) and the University of Las Vegas Nevada (UNLV) have uncovered a genetic link between autism spectrum disorder (ASD) and a rare genetic condition ...

Researchers developed molecules, called splice-switching oligonucleotides (SSOs) that bind to the RNA molecules encoding a protein known as REST. In neuroendocrine tumors, these RNAs are incorrectly ...

A new research paper featured as the cover of Volume 17, Issue 12 of Aging-US was published on December 22, 2025, titled “A ...

Vigeneron GmbH has received IND approval from the FDA for VG-801, a novel mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal dystrophies associated with mutations in the ...

A new research paper featured as the cover of Volume 17, Issue 12 of Aging-US was published on December 22, 2025, titled "A ...

Splicebio S.L. has gained IND clearance from the FDA for its lead program SB-007, a protein splicing gene therapy to address the root genetic cause of Stargardt disease. The company plans to initiate ...

First dose-expansion patient successfully received SB-007 SB-007 addresses the root cause of Stargardt disease with the potential to treat patients across all ABCA4 mutations SB-007 granted FDA ...