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Toward a gene therapy for neurological and somatic MPSIIIA

Virginia Haurigot1, Fatima Bosch1

  • 1Center of Animal Biotechnology and Gene Therapy (CBATEG) and Department of Biochemistry and Molecular Biology; School of Veterinary Medicine; Universitat Autònoma de Barcelona; Barcelona, Spain.

Rare Diseases (Austin, Tex.)
|July 9, 2014
PubMed

Keywords:
AAVCNS gene therapyCSFLSDMPSIIIA

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