Romain Da Costa

Gene Therapeutic Approach Using Mutation-adapted U1 snRNA to Correct a RPGR Splice Defect in Patient-derived Cells

Esther Glaus, F Schmid, Romain Da Costa et al. · Molecular Therapy · 2011 · 66 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17

Molecular analysis of the <i>GNPTAB </i>and <i>GNPTG </i>genes in 13 patients with mucolipidosis type II or type III – identification of eight novel mutations

Marisa Encarnação, Lúcia Lacerda, Romain Da Costa et al. · Clinical Genetics · 2009 · 53 citations

Life Sciences, Molecular Biology, Molecular Genetics +17

Vps13b is required for acrosome biogenesis through functions in Golgi dynamic and membrane trafficking

Romain Da Costa, Morgane Bordessoules, Magali Guilleman et al. · Cellular and Molecular Life Sciences · 2019 · 50 citations · Full text

Life Sciences, Cell Biology, Molecular Biology +12

A Novel Method Combining Vitreous Aspiration and Intravitreal AAV2/8 Injection Results in Retina-Wide Transduction in Adult Mice

Romain Da Costa, Carsten Röger, Jasmin Segelken et al. · Investigative Ophthalmology & Visual Science · 2016 · 31 citations · Full text

Medicine, Molecular Medicine, Vision Science +14

Serpin B1 defect and increased apoptosis of neutrophils in Cohen syndrome neutropenia

Laurence Duplomb, Julie Rivière, Gaëtan Jégo et al. · Journal of Molecular Medicine · 2019 · 25 citations

Life Sciences, Immunology, Molecular Biology +10