La maladie de Crigler-Najjar : un nouveau succès pour la thérapie génique
Nicolas Ferry Ancien directeur de recherche à l’Inserm, Paris, France
Med Sci (Paris) 2023 ; 39 : 833–835
https://www.medecinesciences.org/fr/articles/medsci/full_html/2023/10/msc230215/msc230215.html
Gene Therapy in Patients with the Crigler–Najjar Syndrome
Lorenzo D’Antiga, M.D., Ulrich Beuers, M.D., Giuseppe Ronzitti, Ph.D., Nicola Brunetti-Pierri, M.D., Ulrich Baumann, M.D., Angelo Di Giorgio, M.D., Sem Aronson, Ph.D., Aurelie Hubert, Ph.D., Roberta Romano, M.D., Norman Junge, M.D., Piter Bosma, Ph.D., Giulia Bortolussi, Ph.D., Andrés F. Muro, Ph.D., Ravaka F. Soumoudronga, M.D., Philippe Veron, Ph.D., Fanny Collaud, Ph.D., Nathalie Knuchel-Legendre, M.A.Sc., Philippe Labrune, M.D., and Federico Mingozzi, Ph.D.
N Engl J Med 2023; 389:620-631 DOI: 10.1056/NEJMoa2214084 August 17, 2023
https://www.nejm.org/doi/full/10.1056/NEJMoa2214084?query=featured_home
AAV Immunogenicity : New Answers Create New Questions
Jamie L. Shirley, Roland W. Herzog
Volume 26, ISSUE 11, P2538-2539, November 07, 2018
https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(18)30493-3
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
Amine Meliani, Florence Boisgerault, Romain Hardet, Solenne Marmier, Fanny Collaud, Giuseppe Ronzitti, Christian Leborgne, Helena Costa Verdera, Marcelo Simon Sola, Severine Charles, Alban Vignaud, Laetitia van Wittenberghe, Giorgia Manni, Olivier Christophe, Francesca Fallarino, Christopher Roy, Alicia Michaud, Petr Ilyinskii, Takashi Kei Kishimoto & Federico Mingozzi
Nature Communicationsvolume 9, Article number : 4098 (2018)
Promoterless gene targeting without nucleases rescues lethality of a Crigler‐Najjar syndrome mouse model
Fabiola Porro, Giulia Bortolussi, Adi Barzel, Alessia De Caneva, Alessandra Iaconcig, Simone Vodret, Lorena Zentilin, Mark A Kay, and Andrés F Muro
EMBO Mol Med. 2017 Oct ; 9(10) : 1346–1355. Published online 2017 Jul 27.
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, Fanny Collaud, Severine Charles, Christian Leborgne, Patrice Vidal, Samia Martin, Bernard Gjata, Marcelo Simon Sola, Laetitia van Wittenberghe, Alban Vignaud, Philippe Veron, Piter J Bosma, Andres F Muro, and Federico Mingozzi
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Essai clinique de phase I/II en thérapie cellulaire
Promethera Biosciences annonce le démarrage en France de son essai clinique en thérapie cellulaire
Version française : http://www.france-biotech.org/promethera-biosciences-annonce-le-demarrage-en-france-de-son-essai-clinique-en-therapie-cellulaire/
English version : http://www.news-medical.net/news/20130301/Urea-cycle-disorder-patient-successfully-treated-by-Promethera(R)-HepaStem-in-Phase-III-trial-in-France.aspx
Recommendations for pregnancies in patients with crigler-najjar syndrome.
Wilson JH, Sinaasappel M, Lotgering FK, Langendonk JG.
Departement of internal médicine, internal médicine, metabolic diseases Office D416 Erasmus MC, 2040, 3000 CA, Rotterdam, The Netherlands.
Ezetimibe : A biomarker for efficacy of liver directed UGT1A1 gene therapy for inherited hyperbilirubinemia
Montenegro-Miranda PS, Sneitz N, de Waart DR, Ten Bloemendaal L, Duijst S, de Knegt RJ, Beuers U, Finel M, Bosma PJ.
Tytgat Institute for Intestinal and Liver Research, Academic Medical Center of the University of Amsterdam, Meibergdreef 1105BK, Amsterdam, The Netherlands.
Mycophenolate Mofetil impairs transduction of single-stranded adeno-associated viral vectors.
Montenegro-Miranda P, Ten Bloemendaal L, Kunne C, de Waart DR, Bosma PJ.
Hum Gene Ther. 2011 Jan 11. [Epub ahead of print]
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La Maladie De Crigler Najjar De Type I En Tunisie : Etude de 30 Observations
hajer Aloulou, Afef Ben Thabet, Sonia Khanfir, Lamia Ben Mansour, Imen Chabchoub, Phillipe Labrune, Thouraya Kammoun, Mongia Hachicha
La tunisie Medicale – 2010 ; Vol 88 ( n°010 ) : 707 – 709
http://www.latunisiemedicale.com/article-medicale-tunisie.php?article=1429
Lentiviral vectors that express UGT1A1 in liver and contain miR-142 target sequences normalize hyperbilirubinemia in Gunn rats.
Schmitt F, Remy S, Dariel A, Flageul M, Pichard V, Boni S, Usal C, Myara A, Laplanche S, Anegon I, Labrune P, Podevin G, Ferry N, Nguyen TH.
Gastroenterology. 2010 Sep ;139(3):999-1007, 1007.e1-2. Epub 2010 Jun 19.
Acceleration of the gastrointestinal transit by polyethylene glycol effectively treats unconjugated hyperbilirubinaemia in Gunn rats.
Cuperus FJ, Iemhoff AA, van der Wulp M, Havinga R, Verkade HJ.
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Management of hyperbilirubinemia and prevention of kernicterus in 20 patients with Crigler-Najjar disease
Kevin A. Strauss, Donna L. Robinson, Hendrik J. Vreman, Erik G. Puffenberger, Graham Hart, D. Holmes Morton
Eur J Pediatr – DOI 10.1007/s00431-005-0055-2 – Received : 29 August 2005 / Accepted : 15 November 2005 © Springer-Verlag 2005
http://xa.yimg.com/kq/groups/8696913/206525069/name/CND_Strauss%20EJP%202006.pdf
Successful pregnancy in a crigler-najjar type I patient treated by phototherapy and semimonthly albumin infusions
GAJDOS Vincent ; PETIT Francois ; TRIOCHE Pascale ; MOLLET-BOUDJEMLINE Alix ; CHAUVEAUD Aurelia ; MYARA Anne ; TRIVIN Francois ; FRANCOUAL Jeanne ; LABRUNE Philippe ;
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Prolonged Survival in Three Brothers with Severe Type 2 Crigler-Najjar Syndrome
J.L. Gollan, M.D., M.R.A.C.P., M.R.C.P.**, S.N. Huang, M.D., F.R.C.P.(C) , Barbara Billing, Ph.D., F.R.C.Path.’Correspondence information about the author Ph.D., F.R.C.Path. Barbara Billing , Sheila Sherlock, M.D., F.R.C.P., F.A.C.P.
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