Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

The frontiers of precision medicine have been revolutionized by the development of
Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR)/Cas9 as an editing …

Autosomal recessive genetic forms (DFNB) account for most cases of profound congenital
deafness. Adeno-associated virus (AAV)-based gene therapy is a promising therapeutic …

First worldwide family litigation filed litigation Critical https://patents. darts-ip. com/? family=
49883289&utm_source= google_patent&utm_medium= platform_link&utm_campaign …

Usher syndrome (USH), clinically and genetically heterogeneous, is the leading genetic
cause of combined hearing and vision loss. USH is classified into three types, based on the …

Retinal neurodegeneration associated with the dysfunction or death of photoreceptors is a
major cause of incurable vision loss. Tremendous progress has been made over the last two …

First worldwide family litigation filed litigation Critical https://patents. darts-ip. com/? family=
52105121&utm_source= google_patent&utm_medium= platform_link&utm_campaign …

First worldwide family litigation filed litigation Critical https://patents. darts-ip. com/? family=
49883289&utm_source= google_patent&utm_medium= platform_link&utm_campaign …

2020-03-09 Assigned to NATIONAL INSTITUTES OF HEALTH (NIH), US DEPT. OF HEALTH
AND HUMAN SERVICES (DHHS), US GOVERNMENT reassignment NATIONAL …

Leber congenital amaurosis (LCA) is a severe disorder resulting in visual impairment
usually starting in the first year of life. The most frequent genetic cause of LCA is an intronic …