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Human trials using CRISPR to cure genetically inherited blindness

There are at least 13 types of LCA - this trial is aimed at treating LCA 10

30-Jul-2019

Key points from article :

Heritable disease Leber congenital amaurosis(LCA) causes complete loss of vision.

Will provide a health version of a gene that patients lack through a subretinal injection.

This will be done using the CRISPR gene editing technology.

But this injected gene will not pass into future generations.

Up to 18 people from age 3 onwards will take part in this clinical trial in US.

These trials will be conducted by the pharmaceutical companies Editas Medicine and Allergan.

Mentioned in this article:

Click on resource name for more details.

Allergan

Pharmaceutical company that acquires, develops, and markets brand name drugs.

Editas Medicine

Genome editing company.

Topics mentioned on this page:
Gene Therapy, Vision (health)