The present invention provides DNAzymes for reduction in the expression of the target gene implicated in disease. The present invention also provides a platform technology for the use of DNAzymes to create a reduction in gene expression in zebrafish embryos. More particularly, the present invention describes DNAzyme mediated knockdown of Fmr1 mRNA, gcdh mRNA and gba1mRNA in zebrafish embryos. The present invention also provides a method of creating a model of Fragile X syndrome in Zebrafish embryos.

The present invention relates to a method for the production of n-butanol using a transgenic cell with heterologous expression of 2-hydroxyglutarate dehydrogenase, glutaconate-CoA transferase, (R)-2-hydroxyglutaryl-CoA dehydrogenase, glutaryl CoA dehydrogenase, trans-2-enoyl-CoA reductase (NAD+) and bifunctional aldehyde/alcohol dehydrogenase (NAD+).

Addiction to particular metabolic pathways is common to tumor cells. Disclosed herein are compounds and methods to modulate metabolic signaling facilitated by a glutaryl-CoA dehydrogenase (GCDH) protein for the treatment of cancer. In some embodiments, the compounds can reduce GCDH protein expression or activity and reduce the progression of cancers expressing GCDH.

The present disclosure relates to codon-optimized sequences coding for hGCDH polypeptide and recombinant adeno-associated virus (rAAV) vectors comprising one of said sequences under the control of a promoter component. Also provided herein are viral particles comprising the rAAV vector, a pharmaceutical composition comprising the rAAV vector or the viral particles, and uses thereof in treating Glutaric aciduria type I (GA-I).

The present disclosure relates to codon-optimized sequences coding for hGCDH polypeptide and recombinant adeno-associated virus (rAAV) vectors comprising one of said sequences under the control of a promoter component. Also provided herein are viral particles comprising the rAAV vector, a pharmaceutical composition comprising the rAAV vector or the viral particles, and uses thereof in treating Glutaric aciduria type I (GA-I).