Gene Editing for Exon 51: Why CRISPR Snipping might be better than Exon Skipping for DMD

Gene Editing for Exon 51: Why CRISPR Snipping might be better than Exon Skipping for DMD, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 1: Next Generation Sequencing (NGS)

Gene Editing for Exon 51: Why CRISPR Snipping might be better than Exon Skipping for DMD

Reporter: Aviva Lev-Ari, PhD, RN


UPDATED on 6/19/2018

Sarepta leads off Duchenne MD gene therapy study with a ‘home run’ on efficacy — but can they deliver in 2 years?
john carroll — on June 19, 2018 10:10 AM EDT


UPDATED on 2/27/2017

A CRISPR/Cas9 startup launches on the R&D frontier, working on a one-time fix for Duchenne MD

by john carroll
February 27, 2017 07:29 AM EST


UPDATED on 10/10/2016

The Sarepta dilemma: Bioethics expert Arthur Caplan says it’s time to rethink how to regulate compassion

by john carroll
October 10, 2016 09:43 AM EDT
Updated: 10:56 AM

“Let’s use the Sarepta battle to revisit where we are with compassionate use, what constitutes evidence, what will be accepted as evidence and who pays for collection of the evidence and ultimately early access,” says Caplan





UPDATED on 9/22/2016

On September 19, the FDA okayed eteplirsen to treat Duchenne muscular dystrophy (DMD), a rare genetic disorder that results in muscle degeneration and premature death. Several of its top officials disagreed with the drug’s approval, questioning how beneficial it will be for patients, as ForbesMedPage Today and others reported. In


Amid controversial Sarepta approval decision, FDA head calls for key study retraction



Postmarketing Safety or Effectiveness Data Needed: The 2013 paper was funded by the firm Sarepta Therapeutics, sellers of eteplirsen, a surge in its shares seen after the approval. Eteplirsen will cost patients around $300,000 a year.

Curator: Aviva Lev-Ari, PhD, RN



UPDATED on 4/29/2016

In setback, a majority of FDA experts reject Sarepta’s emotional campaign to gain Duchenne drug OK



UPDATED on 3/25/2016

36 Duchenne experts sign UCLA letter in support of approval of Sarepta’s drug

Mar 21, 2016, 11:47am EDT



Why CRISPR might be better than exon skipping for DMD: Snipping vs. skipping for DMD

By Lauren Martz, Senior Writer
Published on Thursday, January 21, 2016

As if to preempt the regulatory setbacks in Duchenne muscular dystrophy (DMD) that last week disappointed the field, a trio of preclinical studies emerged two weeks earlier showing that cutting out DMD mutations with gene editing might offer a viable alternative to the exon-skipping strategies that have dominated the pipeline. Now, the question is whether there’s reason to believe the mouse studies will translate any better to the clinic.

The studies, published Dec. 31 in Science, provide in vivo proof of concept for the first time that CRISPR-Cas9 used postnatally can have a disease-modifying effect. Despite the hype around its therapeutic promise, the technology has so far proved itself primarily in research applications, for example, in modifying cells for in vitro screening or creating animal models of disease.



Other related articles published in this Open Access Online Scientific Journal include the following:



Gene Editing: The Role of Oligonucleotide Chips

Curators: Larry H Bernstein, MD, FCAP and Aviva Lev-Ari, PhD, RN



Gene Editing by creation of a complement without transcription error

Curator: Larry H. Bernstein, MD, FCAP



UPDATED – Medical Interpretation of the Genomics Frontier – CRISPR – Cas9:  Gene Editing Technology for New Therapeutics

Authors and Curators: Larry H Bernstein, MD, FCAP and Stephen J Williams, PhD and Curator: Aviva Lev-Ari, PhD, RN



DNA Structure and Oligonucleotides

Curator: Larry H Bernstein, MD, FCAP



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