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Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges.
Annarita Miccio , Emmanuelle SIX, Isabelle André, Marina Cavazzana
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Nat Rev Drug Discov2019 Nov 8Pmid / DOI:
30858502 -
A TP63 mutation causes prominent alopecia with mild ectodermal dysplasia.
Alain Hovnanian , Patrick Nitschké, Christine Bole
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J. Invest. Dermatol.2019 Nov 4Pmid / DOI:
31682841 -
Animal models of craniosynostosis.
Laurence Legeai-Mallet , Emilie Dambroise, Nabil Kaci
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Neurochirurgie2019 Nov 1Pmid / DOI:
31563616 -
EFL1 mutations impair eIF6 release to cause Shwachman-Diamond syndrome.
Patrick Revy, Jean-Pierre De Villartay, Christine Bole, Patrick Nitschké
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Blood2019 Nov 1Pmid / DOI:
31151987 -
Candidate Predisposition Variants in Kaposi Sarcoma as Detected by Whole-Genome Sequencing.
Jean-Laurent CASANOVA
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Open Forum Infect Dis2019 Nov 1Pmid / DOI:
31660331 -
Urine-derived cells provide a readily accessible cell type for feeder-free mRNA reprogramming.
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Sci Rep2019 Oct 30Pmid / DOI:
30254308 -
Glibenclamide oral suspension: Suitable and effective in patients with neonatal diabetes.
Michel Polak, Jacques Beltrand
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Pediatr Diabetes2019 Oct 28Pmid / DOI:
30684309 -
Sophie Saunier, Valérie Cormier-Daire, Cécile Jeanpierre, Meriem Garfa-Traoré , Patrick Nitschké
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Hum. Mol. Genet.2019 Oct 27Pmid / DOI:
31042281 -
Alexandre Benmerah
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Hum. Mol. Genet.2019 Oct 27Pmid / DOI:
30388224 -
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Indian J Occup Environ Med2019 Oct 23Pmid / DOI:
31619881 -
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Indian J Occup Environ Med2019 Oct 23Pmid / DOI:
31619881 -
Jean-Laurent CASANOVA
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Curr. Opin. Immunol.2019 Oct 23Pmid / DOI:
31121434 -
Laurent Abel
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Bioinformatics2019 Oct 23Pmid / DOI:
30535305 -
Biallelic mutations in neurofascin cause neurodevelopmental impairment and peripheral demyelination.
Vincent Cantagrel
Source :
Brain2019 Oct 23Pmid / DOI:
31501903
