The advent of clustered regularly interspaced short palindromic repeat (CRISPR) genome
editing, coupled with advances in computing and imaging capabilities, has initiated a new …

The growing understanding of RNA functions and their crucial roles in diseases promotes
the application of various RNAs to selectively function on hitherto “undruggable” proteins …

Background CEP290-associated inherited retinal degeneration causes severe early-onset
vision loss due to pathogenic variants in CEP290. EDIT-101 is a clustered regularly …

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …

Programmable genome integration of large, diverse DNA cargo without DNA repair of
exposed DNA double-strand breaks remains an unsolved challenge in genome editing. We …

Recent advances in the generation, purification and cellular delivery of RNA have enabled
development of RNA-based therapeutics for a broad array of applications. RNA therapeutics …

Gene therapy is the product of man's quest to eliminate diseases. Gene therapy has three
facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where …

CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …