AAV9-CRISPRa-Lama1

AAV9-CRISPRa-Lama1 is an experimental gene therapy platform designed for the treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2-CMD, also known as MDC1A). The therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver CRISPR activation (CRISPRa) components—typically consisting of a catalytically inactive Cas9 (dCas9) fused to transcriptional activators (such as VP64 or miniVPR) and single-guide RNAs (sgRNAs)—to target the promoter region of the LAMA1 gene. By upregulating the expression of LAMA1, which encodes the laminin-111 alpha-1 subunit (a highly homologous compensatory modifier of LAMA2), the therapy aims to compensate for the lack of functional laminin-alpha 2 and restore muscle fiber stability and peripheral nerve myelination. Originally developed by researchers at the Hospital for Sick Children (SickKids) and further optimized at the University of Pittsburgh, this mutation-independent approach is also being commercially advanced by Modalis Therapeutics under the program MDL-101.

Other names

AAV9-CRISPRa-LAMA1AAV-9-CRISPRa-LAMA1AAV 9-CRISPRa-LAMA1AAV9-dCas9-VP64-Lama1AAV-9-dCas9-VP64-Lama1AAV 9-dCas9-VP64-Lama1AAV9-dCas9-miniVPR-Lama1AAV-9-dCas9-miniVPR-Lama1AAV 9-dCas9-miniVPR-Lama1CRISPRa-Lama1CRISPRa-Lama-1CRISPRa-Lama 1