265 related articles for article (PubMed ID: 34943781)
1. Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside.
Cherqui S
Cells; 2021 Nov; 10(12):. PubMed ID: 34943781
[TBL] [Abstract][Full Text] [Related]
2. [Cystinosis: From the gene identification to the first gene therapy clinical trial].
Cherqui S
Med Sci (Paris); 2023 Mar; 39(3):253-261. PubMed ID: 36943122
[TBL] [Abstract][Full Text] [Related]
3. Deficiency of the sedoheptulose kinase (Shpk) does not alter the ability of hematopoietic stem cells to rescue cystinosis in the mouse model.
Goodman S; Khan M; Sharma J; Li Z; Cano J; Castellanos C; Estrada MV; Gertsman I; Cherqui S
Mol Genet Metab; 2021 Dec; 134(4):309-316. PubMed ID: 34823997
[TBL] [Abstract][Full Text] [Related]
4. Is genetic rescue of cystinosis an achievable treatment goal?
Cherqui S
Nephrol Dial Transplant; 2014 Mar; 29(3):522-8. PubMed ID: 23861466
[TBL] [Abstract][Full Text] [Related]
5. Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis.
Harrison F; Yeagy BA; Rocca CJ; Kohn DB; Salomon DR; Cherqui S
Mol Ther; 2013 Feb; 21(2):433-44. PubMed ID: 23089735
[TBL] [Abstract][Full Text] [Related]
6. Allogeneic HSCT transfers wild-type cystinosin to nonhematological epithelial cells in cystinosis: First human report.
Elmonem MA; Veys K; Oliveira Arcolino F; Van Dyck M; Benedetti MC; Diomedi-Camassei F; De Hertogh G; van den Heuvel LP; Renard M; Levtchenko E
Am J Transplant; 2018 Nov; 18(11):2823-2828. PubMed ID: 30030899
[TBL] [Abstract][Full Text] [Related]
7. Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation.
Rocca CJ; Kreymerman A; Ur SN; Frizzi KE; Naphade S; Lau A; Tran T; Calcutt NA; Goldberg JL; Cherqui S
Invest Ophthalmol Vis Sci; 2015 Nov; 56(12):7214-23. PubMed ID: 26540660
[TBL] [Abstract][Full Text] [Related]
8. Hematopoietic Stem Cells Transplantation Can Normalize Thyroid Function in a Cystinosis Mouse Model.
Gaide Chevronnay HP; Janssens V; Van Der Smissen P; Rocca CJ; Liao XH; Refetoff S; Pierreux CE; Cherqui S; Courtoy PJ
Endocrinology; 2016 Apr; 157(4):1363-71. PubMed ID: 26812160
[TBL] [Abstract][Full Text] [Related]
9. Intralysosomal cystine accumulation in mice lacking cystinosin, the protein defective in cystinosis.
Cherqui S; Sevin C; Hamard G; Kalatzis V; Sich M; Pequignot MO; Gogat K; Abitbol M; Broyer M; Gubler MC; Antignac C
Mol Cell Biol; 2002 Nov; 22(21):7622-32. PubMed ID: 12370309
[TBL] [Abstract][Full Text] [Related]
10. Gene transfer may be preventive but not curative for a lysosomal transport disorder.
Hippert C; Dubois G; Morin C; Disson O; Ibanes S; Jacquet C; Schwendener R; Antignac C; Kremer EJ; Kalatzis V
Mol Ther; 2008 Aug; 16(8):1372-81. PubMed ID: 18578013
[TBL] [Abstract][Full Text] [Related]
11. Effects of long-term cysteamine treatment in patients with cystinosis.
Ariceta G; Giordano V; Santos F
Pediatr Nephrol; 2019 Apr; 34(4):571-578. PubMed ID: 29260317
[TBL] [Abstract][Full Text] [Related]
12. Evaluation of the efficacy of cystinosin supplementation through CTNS mRNA delivery in experimental models for cystinosis.
Bondue T; Berlingerio SP; Siegerist F; Sendino-Garví E; Schindler M; Baelde HJ; Cairoli S; Goffredo BM; Arcolino FO; Dieker J; Janssen MJ; Endlich N; Brock R; Gijsbers R; van den Heuvel L; Levtchenko E
Sci Rep; 2023 Nov; 13(1):20961. PubMed ID: 38016974
[TBL] [Abstract][Full Text] [Related]
13. CTNS mRNA molecular analysis revealed a novel mutation in a child with infantile nephropathic cystinosis: a case report.
Papizh S; Serzhanova V; Filatova A; Skoblov M; Tabakov V; van den Heuvel L; Levtchenko E; Prikhodina L
BMC Nephrol; 2019 Oct; 20(1):400. PubMed ID: 31672123
[TBL] [Abstract][Full Text] [Related]
14. Cystinosis and two rare mutations in CTNS gene: two case reports.
Gholami Yarahmadi S; Sarlaki F; Morovvati S
J Med Case Rep; 2022 May; 16(1):181. PubMed ID: 35513889
[TBL] [Abstract][Full Text] [Related]
15. Controversies and research agenda in nephropathic cystinosis: conclusions from a "Kidney Disease: Improving Global Outcomes" (KDIGO) Controversies Conference.
Langman CB; Barshop BA; Deschênes G; Emma F; Goodyer P; Lipkin G; Midgley JP; Ottolenghi C; Servais A; Soliman NA; Thoene JG; Levtchenko EN;
Kidney Int; 2016 Jun; 89(6):1192-203. PubMed ID: 27181776
[TBL] [Abstract][Full Text] [Related]
16. Multisystem involvement, defective lysosomes and impaired autophagy in a novel rat model of nephropathic cystinosis.
Krohn P; Rega LR; Harvent M; Festa BP; Taranta A; Luciani A; Dewulf J; Cremonesi A; Camassei FD; Hanson JVM; Gerth-Kahlert C; Emma F; Berquez M; Devuyst O
Hum Mol Genet; 2022 Jul; 31(13):2262-2278. PubMed ID: 35137071
[TBL] [Abstract][Full Text] [Related]
17. Stem cell microvesicles transfer cystinosin to human cystinotic cells and reduce cystine accumulation in vitro.
Iglesias DM; El-Kares R; Taranta A; Bellomo F; Emma F; Besouw M; Levtchenko E; Toelen J; van den Heuvel L; Chu L; Zhao J; Young YK; Eliopoulos N; Goodyer P
PLoS One; 2012; 7(8):e42840. PubMed ID: 22912749
[TBL] [Abstract][Full Text] [Related]
18. Molecular Mechanisms and Treatment Options of Nephropathic Cystinosis.
Jamalpoor A; Othman A; Levtchenko EN; Masereeuw R; Janssen MJ
Trends Mol Med; 2021 Jul; 27(7):673-686. PubMed ID: 33975805
[TBL] [Abstract][Full Text] [Related]
19. Nephropathic cystinosis: an update on genetic conditioning.
Topaloglu R
Pediatr Nephrol; 2021 Jun; 36(6):1347-1352. PubMed ID: 32564281
[TBL] [Abstract][Full Text] [Related]
20. Potential use of stem cells as a therapy for cystinosis.
Rocca CJ; Cherqui S
Pediatr Nephrol; 2019 Jun; 34(6):965-973. PubMed ID: 29789935
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
