Polycystic kidney disease gene therapy is the best treatment for genetic disease

Polycystic kidney disease gene therapy is the best treatment for genetic disease. However, face considerable obstacles to gene therapy for polycystic kidney disease: First of all, regardless of ADPKD or ARPKD disease gene cDNA fragment large carrier selection and gene into more difficult. Of ADPKD is a focal lesions, cysts occur in only 1% to 5% of the tubular and the presence of some tubular epithelial endocytosis dysfunction, making it difficult to genetic lesions selectively import segment.

 Its genes into the biological effects of PKD1 and PKD2 are widely distributed and participate in a variety of functions in the body. Difficult to estimate. Pritchard the PKDI genes Import PKDI de134 knockout mice avoid PKD1de134/de134 homozygous mice died in the embryonic period but the PKD1 gene into a normal mouse embryo (Es), but caused liver, renal cysts occur. Thivierge, etc. is also observed PKD1 gene overexpression mice develop polycystic kidney disease, and will progress to renal failure. It showed that the PKD1 gene expression or excessive can lead to renal cystic phenotype, the study result, polycystic kidney disease gene replacement therapy into the plight of Shenzhen.

 Later scholars, the direction of gene therapy to block the important function of genes in the onset link. Ricker [Shenzhen to use c.myc antisense oligonucleotides to block the polycystic kidney disease is a very important and significant increase of the proto-oncogene c myc and obviously reduce the cpk mouse kidney weight, slow down the deterioration of renal function. All those who work for polycystic kidney disease gene therapy to make a useful exploration.

In summary polycystic kidney disease gene therapy, gene therapy for polycystic kidney disease is not a realistic way, and multi-drug intervention show slow disease progression in vivo efficacy, but the treatment of validity is still more animal models and randomized controlled clinical studies need to be evaluated. Most of the problems has not been elucidated, the molecular mechanism of ADPKD onset and regulation is very complex, and thus R & D for multiple onset link the different drugs in combination can not only enhance efficacy and reduce adverse reactions, may have a good prospect.