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Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
Massaro, Giulia; Geard, Amy F; Liu, Wenfei; Coombe-Tennant, Oliver; Waddington, Simon N; Baruteau, Julien; Gissen, Paul; Rahim, Ahad A.
Affiliation
  • Massaro G; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Geard AF; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Liu W; Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg 2193, South Africa.
  • Coombe-Tennant O; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Waddington SN; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Baruteau J; Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg 2193, South Africa.
  • Gissen P; Gene Transfer Technology Group, EGA Institute for Women's Health, University College London, London WC1E 6HX, UK.
  • Rahim AA; Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 1EH, UK.
Biomolecules ; 11(4)2021 04 20.
Article in En | MEDLINE | ID: mdl-33924076
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Lysosomal Storage Diseases Limits: Animals / Humans Language: En Journal: Biomolecules Year: 2021 Type: Article Affiliation country: United kingdom
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Lysosomal Storage Diseases Limits: Animals / Humans Language: En Journal: Biomolecules Year: 2021 Type: Article Affiliation country: United kingdom