المؤلفون: Ospedale San Raffaele

المصدر: An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD).

الوصول الحر: https://clinicaltrials.gov/ct2/show/NCT04283227Test

المؤلفون: Ospedale San Raffaele

المصدر: A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)

الوصول الحر: https://clinicaltrials.gov/ct2/show/NCT03392987Test

المؤلفون: Ospedale San Raffaele

المصدر: BfA, Capotondo A, Fasano S, del Carro U, MarchesS, Azuma H, MalagutMC, Amad S, Brambla R, Grompe M, Bordnon C, QuattrA, NaldL. Gene therapy of metachromat leukodystrophy reverses neurologal damage and defs me. J Cl vest. 2006 Nov;116(11):3070-82. do 10.1172/JC8873.
BfA, CesanM, FumagallF, Del Carro U, BaldolC, Canale S, GerevS, Amad S, Falautano M, RovellA, ComG, Roncarolo MG, Sessa M. Metachromat leukodystrophy - mutatn analys proves further evence of genotype-phenotype correlatn. Cl Genet. 2008 Oct;74(4):349-57. do 10.1111/j.1399-0004.2008.01058.x. Epub 2008 Sep 11.
BfA, MontE, LorlL, CesanM, FumagallF, PlatT, BaldolC, Marto S, Calabr A, Canale S, BenedentF, VallantG, Bsco L, Leo S, Kabbara N, ZanettG, Rzo WB, Mehta NA, Calese MP, CasaghM, Boelens JJ, Del Carro U, Dow DJ, Schmt M, AssanellA, Neduva V, DSer C, Stupka E, Gardner J, von Kalle C, Bordnon C, CerF, RovellA, Roncarolo MG, AtA, Sessa M, NaldL. Lental hematopot stem cell gene therapy benefs metachromat leukodystrophy. Scnce. 2013 Aug 23;341(6148):1233158. do 10.1126/scnce.1233158. Epub 2013 Jul 11.
BfA, De Palma M, QuattrA, Del Carro U, Amad S, Vall Sessa M, Fasano S, Brambla R, MarchesS, Bordnon C, NaldL. Correctn of metachromat leukodystrophy the mouse model by transplantatn of genetally modd hematopot stem cells. J Cl vest. 2004 Apr;113(8):1118-29. do 10.1172/JC9205.
Capotondo A, CesanM, Pepe S, Fasano S, GregorS, TononL, VennerMA, Brambla R, QuattrA, Ballab A, Cosma MP, NaldL, BfA. Safety of arylsulfatase A overexpressn for gene therapy of metachromat leukodystrophy. Hum Gene Ther. 2007 Sep;18(9):821-36. do 10.1089/hum.2007.048.
CesanM, Capotondo A, PlatT, SergLS, FumagallF, Roncarolo MG, NaldL, ComG, Sessa M, BfA. Characteratn of new arylsulfatase A gene mutatns reforces genotype-phenotype correlatn metachromat leukodystrophy. Hum Mutat. 2009 Oct;30(10):E936-45. do 10.1002/humu.21093.
A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy

الوصول الحر: https://clinicaltrials.gov/ct2/show/NCT01560182Test