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On April 30th at 13:00 EST the Journal Club will be discussing the recentpaperfrom the Salk Institute. The findings,published in the journal Nature Medicine,showcase a novel CRISPR/Cas9 genome-editing therapy that can suppress the accelerated aging observed in mice with Hutchinson-Gilford progeria syndrome.This treatment provides an important insight into the molecular pathways involved in accelerated aging,as well as how to reduce toxic proteins via gene therapy.The researchers hope to translate this therapy to humans to potentially provide a cure for progeria as well as possibly slowing down the aging process to delay the onset of age-related diseases in everyone.

Abstract

Hutchinson–Gilford progeria syndrome (HGPS) is a rare lethal genetic disorder characterized by symptoms reminiscent of accelerated aging.The major underlying genetic cause is a substitution mutation in the gene coding for lamin A,causing the production of a toxic isoform called progerin.Here we show that reduction of lamin A/progerin by a single-dose systemic administration of adeno-associated virus-delivered CRISPR–Cas9 components suppresses HGPS in a mouse model.

Reference

Beyret,E.,Liao,H.K.,Yamamoto,M.,Hernandez-Benitez,R.,Fu,Y.,Erikson,G.,… & Belmonte,J.C.I.(2019).Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome.Nature medicine,1.

About the author

Dr.Oliver Medvedik

Oliver Medvedik,Co-founder of Genspace citizen science laboratory in Brooklyn NY,earned his Ph.D.at Harvard Medical School in the Biomedical and Biological Sciences program.As part of his doctoral work he has used single-celled budding yeast as a model system to map the genetic pathways that underlie the processes of aging in more complex organisms,such as humans.Prior to arriving in Boston for his doctoral studies,he has lived most of his life in New York City.He obtained his bachelor's degree in biology from Hunter College,City University of New York.Since graduating from Harvard,he has worked as a biotechnology consultant,taught molecular biology to numerous undergraduates at Harvard University and mentored two of Harvard's teams for the international genetically engineered machines competition (IGEM) held annually at M.I.T.
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