Krystal Biotech, Inc.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF AUTOSOMAL RECESSIVE CONGENITAL ICHTHYOSIS

Abstract:

The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding a transglutaminase (TGM) polypeptide (e.g., a Transglutaminase-1 (TGM1) polypeptide); viruses comprising the recombinant nucleic acids; compositions comprising the recombinant nucleic acids and/or viruses; methods of their use; and articles of manufacture or kits thereof.