CRISPR Applications {Vision Possibilities}

 Editas Medicine and Allergan have teamed up in order to find a treatment for Leber Congenital Amaurosis 10(LCA10). LCA 10 is an inherited degenerative eye disorder. LCA10 symptoms become apparent within the first year of birth, LCA10 will continue to worsen throughout the individuals life time, often leaving them severely visually impaired and or blind. LCA10 is considered to be relatively rare affecting 1-2 out of 100,000 births. LCA10 specifically is the worsening of the ocular photoreceptor cells. Editas Medicine and Allergan have announced their clinical trial "The Brilliance Phase 1/2 Trial of AGN-151587(edit 101) for Treatment of LCA10. Their exact description "The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of AGN-151587 (EDIT-101) administered via sub retinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene ("LCA10-IVS26")".  LCR is a promising candidate for CRISPR because the disease is monogenic meaning the involvement or control by a singular gene, making the targeting much easier and quicker. 

Picture below describes the 'protocol' of AGN-151587.

Bibliography 

“Research and Pipeline.” Editas Medicine, 5 Nov. 2020, www.editasmedicine.com/gene-editing-pipeline/.

Park, Alice. “CRISPR Gene Editing Is Being Tested in Human Patients.” Time, Time, 6 Aug. 2019, time.com/5642755/crispr-gene-editing-humans/.

“Allergan and Editas Medicine Initiate the Brilliance Phase 1/2 Clinical Trial of AGN-151587 (EDIT-101) for the Treatment of LCA10.” Editas Medicine, ir.editasmedicine.com/news-releases/news-release-details/allergan-and-editas-medicine-initiate-brilliance-phase-12.

“Single Ascending Dose Study in Participants With LCA10 - Full Text View.” Full Text View - ClinicalTrials.gov, www.clinicaltrials.gov/ct2/show/NCT03872479?cond=LCA10.

“Leber Congenital Amaurosis: MedlinePlus Genetics.” MedlinePlus, U.S. National Library of Medicine, 18 Aug. 2020, medlineplus.gov/genetics/condition/leber-congenital-amaurosis/.