Emory University

Children's Hospital Medical Center

Children's Healthcare of Atlanta, Inc.

Harrison C Brown

Christopher B Doering

H Trent Spencer

Assem G Ziady

Matthew Siefert

Songbai Lin

Trent H Spencer

Disclosed are methods for the enhancement of nucleic acid delivery systems. The methods may employ treatment with a compound and/or an RNAi molecule in combination with a nucleic acid to improve nucleic acid uptake into a cell. In particular, the disclosed methods may be useful for improved gene therapy techniques.

Compositions and methods for nucleic acid transfer

Described are recombinant nucleic acid molecules for increased expression of Cas9 in human liver. In some embodiments, the recombinant nucleic acid molecules are provided in compositions and methods for gene editing, specifically using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR).

Cas9 nucleic acid molecules and their use

Disclosed herein are recombinant viral vectors comprising a liver specific promotor in operable combination with a heterologous nucleic acid sequence encoding a protein, such as a clotting factor. Methods of treating a subject with a clotting disorder, such as hemophilia A or hemophilia B, are also provided.

Recombinant promoters and vectors for protein expression in liver and use thereof

CAS9 nucleic acid molecules and their use

Growing community of inventors

Atlanta, GA, United States of America

Harrison C Brown