918 related articles for article (PubMed ID: 23845948)
1. 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
Science; 2013 Aug; 341(6148):1233158. PubMed ID: 23845948
[TBL] [Abstract][Full Text] [Related]
2. 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
Lancet; 2022 Jan; 399(10322):372-383. PubMed ID: 35065785
[TBL] [Abstract][Full Text] [Related]
3. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Sessa M; Lorioli L; Fumagalli F; Acquati S; Redaelli D; Baldoli C; Canale S; Lopez ID; Morena F; Calabria A; Fiori R; Silvani P; Rancoita PM; Gabaldo M; Benedicenti F; Antonioli G; Assanelli A; Cicalese MP; Del Carro U; Sora MG; Martino S; Quattrini A; Montini E; Di Serio C; Ciceri F; Roncarolo MG; Aiuti A; Naldini L; Biffi A
Lancet; 2016 Jul; 388(10043):476-87. PubMed ID: 27289174
[TBL] [Abstract][Full Text] [Related]
4. Gene therapy in metachromatic leukodystrophy.
Sevin C; Cartier-Lacave N; Aubourg P
Int J Clin Pharmacol Ther; 2009; 47 Suppl 1():S128-31. PubMed ID: 20040324
[TBL] [Abstract][Full Text] [Related]
5. Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy.
Meneghini V; Lattanzi A; Tiradani L; Bravo G; Morena F; Sanvito F; Calabria A; Bringas J; Fisher-Perkins JM; Dufour JP; Baker KC; Doglioni C; Montini E; Bunnell BA; Bankiewicz K; Martino S; Naldini L; Gritti A
EMBO Mol Med; 2016 May; 8(5):489-510. PubMed ID: 27025653
[TBL] [Abstract][Full Text] [Related]
6. In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice.
Consiglio A; Quattrini A; Martino S; Bensadoun JC; Dolcetta D; Trojani A; Benaglia G; Marchesini S; Cestari V; Oliverio A; Bordignon C; Naldini L
Nat Med; 2001 Mar; 7(3):310-6. PubMed ID: 11231629
[TBL] [Abstract][Full Text] [Related]
7. Atidarsagene autotemcel for metachromatic leukodystrophy.
Messina M; Gissen P
Drugs Today (Barc); 2023 Feb; 59(2):63-70. PubMed ID: 36811406
[TBL] [Abstract][Full Text] [Related]
8. Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy.
Capotondo A; Cesani M; Pepe S; Fasano S; Gregori S; Tononi L; Venneri MA; Brambilla R; Quattrini A; Ballabio A; Cosma MP; Naldini L; Biffi A
Hum Gene Ther; 2007 Sep; 18(9):821-36. PubMed ID: 17845130
[TBL] [Abstract][Full Text] [Related]
9. Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy.
Doerr J; Böckenhoff A; Ewald B; Ladewig J; Eckhardt M; Gieselmann V; Matzner U; Brüstle O; Koch P
Mol Ther; 2015 Sep; 23(9):1519-31. PubMed ID: 26061647
[TBL] [Abstract][Full Text] [Related]
10. Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.
Piguet F; Sondhi D; Piraud M; Fouquet F; Hackett NR; Ahouansou O; Vanier MT; Bieche I; Aubourg P; Crystal RG; Cartier N; Sevin C
Hum Gene Ther; 2012 Aug; 23(8):903-14. PubMed ID: 22642214
[TBL] [Abstract][Full Text] [Related]
11. Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.
Sevin C; Benraiss A; Van Dam D; Bonnin D; Nagels G; Verot L; Laurendeau I; Vidaud M; Gieselmann V; Vanier M; De Deyn PP; Aubourg P; Cartier N
Hum Mol Genet; 2006 Jan; 15(1):53-64. PubMed ID: 16311251
[TBL] [Abstract][Full Text] [Related]
12. In vitro correction of ARSA deficiency in human skin fibroblasts from metachromatic leukodystrophy patients after treatment with microencapsulated recombinant cells.
Lagranha VL; Baldo G; de Carvalho TG; Burin M; Saraiva-Pereira ML; Matte U; Giugliani R
Metab Brain Dis; 2008 Dec; 23(4):469-84. PubMed ID: 18797988
[TBL] [Abstract][Full Text] [Related]
13. Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A.
Miyake N; Miyake K; Sakai A; Yamamoto M; Suzuki H; Shimada T
Sci Rep; 2021 Oct; 11(1):20513. PubMed ID: 34654893
[TBL] [Abstract][Full Text] [Related]
14. Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.
Biffi A; Capotondo A; Fasano S; del Carro U; Marchesini S; Azuma H; Malaguti MC; Amadio S; Brambilla R; Grompe M; Bordignon C; Quattrini A; Naldini L
J Clin Invest; 2006 Nov; 116(11):3070-82. PubMed ID: 17080200
[TBL] [Abstract][Full Text] [Related]
15. Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment.
Wu THY; Brown HA; Church HJ; Kershaw CJ; Hutton R; Egerton C; Cooper J; Tylee K; Cohen RN; Gokhale D; Ram D; Morton G; Henderson M; Bigger BW; Jones SA
Mol Genet Metab; 2024 May; 142(1):108349. PubMed ID: 38458124
[TBL] [Abstract][Full Text] [Related]
16. Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.
Meneghini V; Frati G; Sala D; De Cicco S; Luciani M; Cavazzin C; Paulis M; Mentzen W; Morena F; Giannelli S; Sanvito F; Villa A; Bulfone A; Broccoli V; Martino S; Gritti A
Stem Cells Transl Med; 2017 Feb; 6(2):352-368. PubMed ID: 28191778
[TBL] [Abstract][Full Text] [Related]
17. Broadening the indications for hematopoietic stem cell genetic therapies.
Williams DA
Cell Stem Cell; 2013 Sep; 13(3):263-4. PubMed ID: 24012366
[TBL] [Abstract][Full Text] [Related]
18. Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery.
Luca T; Givogri MI; Perani L; Galbiati F; Follenzi A; Naldini L; Bongarzone ER
Mol Ther; 2005 Oct; 12(4):669-79. PubMed ID: 16087406
[TBL] [Abstract][Full Text] [Related]
19. Developing therapeutic approaches for metachromatic leukodystrophy.
Patil SA; Maegawa GH
Drug Des Devel Ther; 2013; 7():729-45. PubMed ID: 23966770
[TBL] [Abstract][Full Text] [Related]
20. Understanding caregiver descriptions of initial signs and symptoms to improve diagnosis of metachromatic leukodystrophy.
Eichler F; Sevin C; Barth M; Pang F; Howie K; Walz M; Wilds A; Calcagni C; Chanson C; Campbell L
Orphanet J Rare Dis; 2022 Oct; 17(1):370. PubMed ID: 36195888
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
