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GENE SILENCING THERAPEUTICS IN NEURO-DEGENERATIVE DISEASES: A REVIEW. Parmpreet Singh, Sarabjit Singh, Dinesh Kumar, Pooja Sharma, Harwinder Singh Rao

GENE SILENCING THERAPEUTICS IN NEURO-DEGENERATIVE DISEASES: A REVIEW.

Parmpreet Singh, Sarabjit Singh, Dinesh Kumar, Pooja Sharma, Harwinder Singh Rao

International Journal of Natural Product Science 2012: Spl Issue 1:202.

Abstract(RBIP-202)

Gene silencing is iRNA (interfering Ribonucleic Acid) therapeutics. The technology is based on small interfering RNA (siRNA), offers a powerful and highly specific tool for therapeutic gene silencing. Many diseases like Neurodegenerative disorders (Parkinsonism disease, Huntington’s disease, Alzheimer disease, Epilepsy etc.), Tumors, Retinal diseases are treated by gene silencing technology. Huntington’s disease possesses highest prevalence of all autosomal CAG repeat neurodegenerative diseases; confer toxicity to the mutant protein (Huntington’s protein). Patient with Huntington develops involuntary movements, behavior changes in 4th decade of life. Disease modifying therapy is used to counter toxic effects of mutant & cellular replacement. It can be done with the help of oligonulcleotide approach to suppress genes such as iRNA & ASO’s (antisense oligonucleotides). RNA and ASO’s provide approaches towards disease modifications by their unique, highly efficient and specific mechanism of action.

Keywords: Gene silencing, Huntington’s, oligonulcleotide, Neurodegenerative.
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