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Academic Journal

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

Subjects: AAV; Mouse model; Muscle

  • Source: ISSN: 2352-3964 ; EBioMedicine ; https://hal.sorbonne-universite.fr/hal-03163331 ; EBioMedicine, 2020, 61, pp.103052. ⟨10.1016/j.ebiom.2020.103052⟩.

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Academic Journal

A retrosynthetic biology approach to therapeutics: from conception to delivery

Subjects: prediction; homeostasis; optimization

  • Source: ISSN: 0958-1669 ; Current Opinion in Biotechnology ; https://hal.inrae.fr/hal-02651873 ; Current Opinion in Biotechnology, 2012, 23 (6), pp.948-956. ⟨10.1016/j.copbio.2012.03.009⟩ ;

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