Bayer Healthcare LLC

Crispr Therapeutics Ag

Other

Alan Richard Brooks

Richard L Feldman

Shaun M Lippow

Peter Bringmann

Shujun Yuan

Jian-Ming Gu

Karen Vo

Provided include compositions, methods, and systems for modulating the expression, function, and/or activity of a target gene, for example a blood-clotting protein such as Factor VIII (FVIII), in a cell by genome editing. Also provided include compositions, methods, and systems for treating a subject having or suspected of having a disorder or health condition, e.g., Hemophilia A, employing ex vivo and/or in vivo genome editing.

Compositions and methods for gene editing by targeting transferrin

Provided herein, in some embodiments, are materials and methods for treating hemophilia A in a subject ex vivo or in vivo. Also provided herein, in some embodiments, are materials and methods for knocking in a coding sequence encoding a synthetic FVIII having a B domain substitute into a genome.

Gene editing for hemophilia A with improved factor VIII expression

Targeted Factor VIII molecules comprising a Factor VIII linked with at least one domain that specifically binds to a membrane protein on a red blood cell is provided. The disclosed targeted coagulation factors prolong their duration of action and thus, are an improvement for the treatment of hematological diseases such as hemophilia A.

Red blood cell targeted factor VIII and method of using the same

A concrete basement stairway is created, by positioning a stairway formbetween a first pair of concrete receiving foundation channelsand a second pair of concrete receiving foundation channelsof any size foundation, the stairway form including a single piece stairstep panel, coupled by pivotable latching members, between a pair of parallel inner stairway side panelsand, the inner stairway side panels configured to permit concrete to flow between the two pairs of concrete receiving foundation channels and spaces beneath the stairstep panel, in a manner forming a series of solidified concrete steps unified, and thus leak proof, with concrete solidified within the concrete receiving foundation channels. The stairway is formed simultaneously with the foundation so that a builder can complete the job without awaiting shipment of a pre-cast stairway requiring returning to the site. Reusable lightweight aluminum forms are employed for easy handling.

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Cambridge, MA, United States of America

Alan Richard Brooks