AAV vector-mediated deletion of large mutational hotspot for treatment of DUCHENNE muscular dystrophy

Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.

Saved in:
Bibliographic Details
Main Authors BULAKLAK, KAREN, GERSBACH, CHARLES A, ROBINSON-HAMM, JACQUELINE N
Format Patent
LanguageChinese
English
Published 01.02.2021
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
Online AccessGet full text

Cover

More Information
Summary:Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.
Bibliography:Application Number: TW20209112555