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Quoted phrase not found in phrase index: "Metachromatic leukodystrophy, adult type"
Page 1
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Fumagalli F, Calbi V, Natali Sora MG, Sessa M, Baldoli C, Rancoita PMV, Ciotti F, Sarzana M, Fraschini M, Zambon AA, Acquati S, Redaelli D, Attanasio V, Miglietta S, De Mattia F, Barzaghi F, Ferrua F, Migliavacca M, Tucci F, Gallo V, Del Carro U, Canale S, Spiga I, Lorioli L, Recupero S, Fratini ES, Morena F, Silvani P, Calvi MR, Facchini M, Locatelli S, Corti A, Zancan S, Antonioli G, Farinelli G, Gabaldo M, Garcia-Segovia J, Schwab LC, Downey GF, Filippi M, Cicalese MP, Martino S, Di Serio C, Ciceri F, Bernardo ME, Naldini L, Biffi A, Aiuti A. Fumagalli F, et al. Lancet. 2022 Jan 22;399(10322):372-383. doi: 10.1016/S0140-6736(21)02017-1. Lancet. 2022. PMID: 35065785 Free PMC article. Clinical Trial.
BACKGROUND: Effective treatment for metachromatic leukodystrophy (MLD) remains a substantial unmet medical need. ...METHODS: This study is an integrated analysis of results from a prospective, non-randomised, phase 1/2 clinical study and expanded-acces …
BACKGROUND: Effective treatment for metachromatic leukodystrophy (MLD) remains a substantial unmet medical need. ...METHODS: T …
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, Zanetti G, Rizzo WB, Mehta NA, Cicalese MP, Casiraghi M, Boelens JJ, Del Carro U, Dow DJ, Schmidt M, Assanelli A, Neduva V, Di Serio C, Stupka E, Gardner J, von Kalle C, Bordignon C, Ciceri F, Rovelli A, Roncarolo MG, Aiuti A, Sessa M, Naldini L. Biffi A, et al. Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11. Science. 2013. PMID: 23845948 Clinical Trial.
Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disease caused by arylsulfatase A (ARSA) deficiency. ...
Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disease caused by arylsulfatase A (ARSA) deficiency. ...
Recent Advancements in the Diagnosis and Treatment of Leukodystrophies.
Bradbury AM, Ream MA. Bradbury AM, et al. Semin Pediatr Neurol. 2021 Apr;37:100876. doi: 10.1016/j.spen.2021.100876. Epub 2021 Feb 10. Semin Pediatr Neurol. 2021. PMID: 33892849 Review.
Transduction of autologous hematopoietic stem cells with lentiviral vectors, referred to as ex vivo gene therapy, circumvents some, but not all, of the risks of traditional transplantation and has recently been demonstrated to be safe and efficective in clinical studies of …
Transduction of autologous hematopoietic stem cells with lentiviral vectors, referred to as ex vivo gene therapy, circumvents some, but not …
Gene-Based Approaches to Inherited Neurometabolic Diseases.
Poletti V, Biffi A. Poletti V, et al. Hum Gene Ther. 2019 Oct;30(10):1222-1235. doi: 10.1089/hum.2019.190. Epub 2019 Sep 10. Hum Gene Ther. 2019. PMID: 31397176 Review.
These efforts were successfully coupled with unprecedented clinical results of the trials employing the newly developed technology and with the novel establishment of academic-industrial partnerships. ...This review provides an overview of the GT strategies currently under …
These efforts were successfully coupled with unprecedented clinical results of the trials employing the newly developed technology an …
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.
Babcock MC, Mikulka CR, Wang B, Chandriani S, Chandra S, Xu Y, Webster K, Feng Y, Nelvagal HR, Giaramita A, Yip BK, Lo M, Jiang X, Chao Q, Woloszynek JC, Shen Y, Bhagwat S, Sands MS, Crawford BE. Babcock MC, et al. Sci Rep. 2021 Jul 14;11(1):14486. doi: 10.1038/s41598-021-93601-1. Sci Rep. 2021. PMID: 34262084 Free PMC article.
Krabbe disease (KD) and metachromatic leukodystrophy (MLD) are caused by accumulation of the glycolipids galactosylceramide (GalCer) and sulfatide and their toxic metabolites psychosine and lysosulfatide, respectively. ...Despite the significant benefits observed in …
Krabbe disease (KD) and metachromatic leukodystrophy (MLD) are caused by accumulation of the glycolipids galactosylceramide (G …
Gene therapy of metachromatic leukodystrophy.
Matzner U, Gieselmann V. Matzner U, et al. Expert Opin Biol Ther. 2005 Jan;5(1):55-65. doi: 10.1517/14712598.5.1.55. Expert Opin Biol Ther. 2005. PMID: 15709909 Review.
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease that is caused by a deficiency of arylsulfatase A (ASA). ...Following transplantation of bone marrow cells overexpressing ASA from a retroviral vector, donor-type cells secrete ASA, which is en …
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease that is caused by a deficiency of arylsulfatase A (ASA). ... …
Neurodegenerative disease after hematopoietic stem cell transplantation in metachromatic leukodystrophy.
Al-Saady M, Beerepoot S, Plug BC, Breur M, Galabova H, Pouwels PJW, Boelens JJ, Lindemans C, van Hasselt PM, Matzner U, Vanderver A, Bugiani M, van der Knaap MS, Wolf NI. Al-Saady M, et al. Ann Clin Transl Neurol. 2023 Jul;10(7):1146-1159. doi: 10.1002/acn3.51796. Epub 2023 May 22. Ann Clin Transl Neurol. 2023. PMID: 37212343 Free PMC article.
OBJECTIVE: Metachromatic leukodystrophy is a lysosomal storage disease caused by deficient arylsulfatase A. ...METHODS: Three metachromatic leukodystrophy patients treated with hematopoietic stem cell transplantation with a progressive clinical …
OBJECTIVE: Metachromatic leukodystrophy is a lysosomal storage disease caused by deficient arylsulfatase A. ...METHODS: Three …
Immune response in leukodystrophies.
Eichler F, Van Haren K. Eichler F, et al. Pediatr Neurol. 2007 Oct;37(4):235-44. doi: 10.1016/j.pediatrneurol.2007.06.011. Pediatr Neurol. 2007. PMID: 17903666 Review.
In this review, we compare three common leukodystrophies, each with a different immune response: (1) X-linked adrenoleukodystrophy, which demonstrates a severe, lymphocytic inflammatory response; (2) metachromatic leukodystrophy, which yields a histiocytic response; …
In this review, we compare three common leukodystrophies, each with a different immune response: (1) X-linked adrenoleukodystrophy, which de …
Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knock-Out Mice.
St Martin T, Seabrook TA, Gall K, Newman J, Avila N, Hayes A, Kivaa M, Lotterhand J, Mercaldi M, Patel K, Rivas IJ, Woodcock S, Wright TL, Seymour AB, Francone OL, Gingras J. St Martin T, et al. J Neurosci. 2023 May 10;43(19):3567-3581. doi: 10.1523/JNEUROSCI.1829-22.2023. Epub 2023 Mar 28. J Neurosci. 2023. PMID: 36977578 Free PMC article.
Metachromatic leukodystrophy (MLD) is a rare, inherited, demyelinating lysosomal storage disorder caused by mutations in the arylsulfatase-A gene (ARSA). ...Together, these findings support the use of intravenous delivery of HSC15/ARSA-mediated gene therapy for the …
Metachromatic leukodystrophy (MLD) is a rare, inherited, demyelinating lysosomal storage disorder caused by mutations in the a …
Advances and pitfalls of cell therapy in metabolic leukodystrophies.
Miranda CO, Brites P, Mendes Sousa M, Teixeira CA. Miranda CO, et al. Cell Transplant. 2013;22(2):189-204. doi: 10.3727/096368912X656117. Epub 2012 Sep 21. Cell Transplant. 2013. PMID: 23006656 Free article. Review.
Recently, interest on cell therapy has increased, and the leukodystrophies for which metabolic correction is needed have become first-choice candidates for cell-based clinical trials. In this review, we present and discuss the available cell transplantation therapies in me …
Recently, interest on cell therapy has increased, and the leukodystrophies for which metabolic correction is needed have become first-choice …
114 results