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View article: Ex vivo gene editing and cell therapy for hereditary tyrosinemia type 1
Ex vivo gene editing and cell therapy for hereditary tyrosinemia type 1 Open
Background: We previously demonstrated the successful use of in vivo CRISPR gene editing to delete 4-hydroxyphenylpyruvate dioxygenase ( HPD ) to rescue mice deficient in fumarylacetoacetate hydrolase (FAH), a disorder known as hereditary …
View article: EX VIVO GENE EDITING AND CELL THERAPY FOR HEREDITARY TYROSINEMIA TYPE 1
EX VIVO GENE EDITING AND CELL THERAPY FOR HEREDITARY TYROSINEMIA TYPE 1 Open
Background & Aims We previously demonstrated the successful use of in vivo CRISPR gene editing to delete 4-hydroxyphenylpyruvate dioxygenase ( HPD ) to rescue mice deficient in fumarylacetoacetate hydrolase (FAH), a disorder known as hered…
View article: Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins Results in High Levels of Gene Editing in Primary Hepatocytes
Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins Results in High Levels of Gene Editing in Primary Hepatocytes Open
Adeno-associated virus vectors are the most used delivery method for liver-directed gene editing. Still, they are associated with significant disadvantages that can compromise the safety and efficacy of therapies. Here, we investigate the …
View article: Delivery Approaches for Therapeutic Genome Editing and Challenges
Delivery Approaches for Therapeutic Genome Editing and Challenges Open
Impressive therapeutic advances have been possible through the advent of zinc-finger nucleases and transcription activator-like effector nucleases. However, discovery of the more efficient and highly tailorable clustered regularly interspa…