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Type I CRISPR-Cas expands the human genome engineering toolkit
Unmet Need Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Systems (Cas) provide immune protection to bacteria and archaea. CRISPR-Cas systems can be mono-subunit (Class 2) or multi-subunit (Class 1). Class 2 CRISPR-Cas systems, especially Type…
Perfusate for optimized gene delivery to donor organs undergoing ex vivo machine perfusion
Unmet Need Effective gene delivery is notoriously difficult to achieve to an entire target organ. Gene therapies have traditionally been administered intravascularly, requiring high vector doses to attain therapeutic levels of expression. Because of this…
An injectable hydrogel scaffold that delivers gene therapies locally
Unmet Need Gene therapy has applications ranging from tissue engineering to vaccine development to infectious disease. Most approaches to deliver nucleic material involve the systemic intravenous delivery of condensed nucleic acid (e.g., within a virus…