Antisense Oligonucleotides (ASOs) Products for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive disease characterized by loss of spinal cord motor neurons, muscle atrophy and infantile death or severe disability. In the majority of patients with SMA, the gene encoding survival motor neuron 1 (SMN1) is deleted or nonfunctional. The antisense oligonucleotides (ASOs) have been developed in the correction of aberrant RNA splicing characteristic of SMA. Currently, a number of cis-acting splice modifiers have been found in the region of exon 7, the steric block of which enhances the retention of the exon and a resultant full-length mRNA sequence. Therefore, the ASO products targeted to these splice motifs are able to change the splicing of SMN1 and produce more functional SMN protein.

Creative Biolabs is a world-renowned service provider for antisense oligonucleotides therapy development. With years of experience, we provide a variety of top-quality ASO products for HIV/AIDS therapy. A series of stringent criteria have been applied in quality control of antisense products to guarantee reliability. We are dedicated to providing the best-characterized antisense therapy service to help our customers all over the world. Please feel free to contact us by e-mail for a quote.

Reference

1. Wood, M.; et al. (2017). Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape. Human Molecular Genetics. 26(R2), pp. R151-R159.