Using CRISPR to Identify Novel Therapeutic Targets in FSHD

CRISPR-Cas9, originally discovered in prokaryotic organisms to be part of their immune system, has rapidly developed into a gene editing tool far more accurate than its predecessors. This technology is thus generating a lot of buzz due to its potential to correct genetic diseases. However, what if it could be used to identify factors that can lessen disease severity? These targets could be the source of novel treatments. A recent study out in Science Translational Medicine led by Angela Lek PhD and Louis Kunkel PhD explored this in Facioscapulohumeral Muscular Dystrophy (FSHD), a type of muscular dystrophy for which there is no treatment or cure.  

FSHD News: Bonita’s Ray wins scholastic essay contest

Expressing his hardships and triumphs while battling facioscapulohumeral muscular dystrophy, Bonita eighth-grader Sam Ray is one of the winners of the national Scholastic’s annual essay contest.The annual essay, “Breaking Barriers: In Sports, In Life,” was created by Scholastic and Major Baseball League in honor of Jackie Robinson, to inspire students by Robinson’s perseverance and to teach them how to use the same values he did to overcome hardships within their lives.