Spark Therapeutics, Inc.

Sorbonne Université

Genethon

Universite Paris Cité

Inserm

Sean Armour

Xavier Anguela

Federico Mingozzi

Jayme Nordin

Christian Leborgne

Jordan Dimitrov

Sébastien Lacroix-desmazes

The invention provides nucleic acids encoding acid α-glucosidase (GAA). In certain embodiments, nucleic acids have greater than about 86% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs:1-5. In certain embodiments, nucleic acids encoding acid α-glucosidase (GAA) contain less than 127 CpG dinucleotides. Expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids encoding acid α-glucosidase (GAA) are also provided.

Codon-optimized acid alpha-glucosidase expression cassettes and methods of using same

Growing community of inventors

Holland, PA, United States of America

Sean Armour