bluebird bio, Inc.
PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE

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Abstract:

The present disclosure provides improved genome editing compositions and methods for editing a PCSK9 gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of hypercholesterolemia or a condition associated therewith.

Status:
Application
Type:

Utility

Filling date:

4 Oct 2018

Issue date:

3 Dec 2020