Fetal gene therapy for neurodegenerative lysosomal storage diseases

J Inherit Metab Dis. 2019 May;42(3):391-393. doi: 10.1002/jimd.12018. Epub 2019 Feb 4.

Julien Baruteau^{1

2

3}, Simon N Waddington^{1

4}

¹ Gene Transfer Technology Group, Institute for Women's Health, University College London, London, UK.
² Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
³ Genetics and Genomic Medicine Programme, Great Ormond Street Institute of Child Health, University College London, London, UK.
⁴ Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.

No abstract available

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