Trans-splicing is a unique process in gene expression where a short sequence, known as a spliced leader (SL), is added to the 5' end of mRNA molecules. This mechanism is particularly significant in ...

University of Zurich scientists demonstrated that they could reassemble and express large genes using a new dual adeno-associated viral (AAV) vector technology that depends on mRNA trans-splicing.

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 ...

It has been proposed that isoforms of Drosophila mod(mdg4) result from the trans-splicing of independent mRNA transcripts derived from both anti-parallel DNA strands. In the August 14 Proceedings of ...

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 ...

Cell and gene therapies hold tremendous value due to their efficacy and potency in a diverse range of indications. The classical approach to gene therapy involves the replacement of a defective gene ...

Range of Methods Used in Generating and Purifying AAV Vectors Vectors derived from adeno-associated virus (AAV) provide promising gene delivery vehicles that can be used effectively in large-scale ...

Splice-splicing oligonucleotides targeting amyloid proteins that can reduce brain plaques in Alzheimer's mice Spliceosome-mediated RNA trans-splicing (SMaRT) -- gene reprogramming system designed to ...

Alternative splicing, a clever way a cell generates many different variations of messenger RNAs - single-stranded RNAs involved in protein synthesis - and proteins from the same stretch of DNA, plays ...