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Page 1
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inherited human diseases, offering durable and possibly curative clinical benefit with a single treatment. This review is limited to gene …
For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inher …
Retinal gene therapy.
Kumaran N, Michaelides M, Smith AJ, Ali RR, Bainbridge JWB. Kumaran N, et al. Br Med Bull. 2018 Jun 1;126(1):13-25. doi: 10.1093/bmb/ldy005. Br Med Bull. 2018. PMID: 29506236 Review.
AREAS OF AGREEMENT: Gene therapy can improve vision in RPE65-associated Leber Congenital Amaurosis (RPE65-LCA). Potential benefit depends on efficient gene transfer and is limited by the extent of retinal degeneration. ...AREAS TIMELY FOR DEVELOPING RESEARCH: …
AREAS OF AGREEMENT: Gene therapy can improve vision in RPE65-associated Leber Congenital Amaurosis (RPE65-LCA). Potenti …
Human Cardiac Gene Therapy.
Ishikawa K, Weber T, Hajjar RJ. Ishikawa K, et al. Circ Res. 2018 Aug 17;123(5):601-613. doi: 10.1161/CIRCRESAHA.118.311587. Circ Res. 2018. PMID: 30355138 Free PMC article. Review.
In the past 10 years, there has been tremendous progress made in the field of gene therapy. Effective treatments of Leber congenital amaurosis, hemophilia, and spinal muscular atrophy have been largely based on the efficiency and safety of adeno-associated ve …
In the past 10 years, there has been tremendous progress made in the field of gene therapy. Effective treatments of Leber congenit …
Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials.
Daich Varela M, Cabral de Guimaraes TA, Georgiou M, Michaelides M. Daich Varela M, et al. Br J Ophthalmol. 2022 Apr;106(4):445-451. doi: 10.1136/bjophthalmol-2020-318483. Epub 2021 Mar 12. Br J Ophthalmol. 2022. PMID: 33712480 Free PMC article. Review.
Leber congenital amaurosis (LCA) is a severe congenital/early-onset retinal dystrophy. ...The next 5 years are likely to be key to whether genetic therapies will achieve their full promise, and whether stem cell/cellular therapies will break through into c …
Leber congenital amaurosis (LCA) is a severe congenital/early-onset retinal dystrophy. ...The next 5 years are likely t …
Gene therapy for RPE65-related retinal disease.
Miraldi Utz V, Coussa RG, Antaki F, Traboulsi EI. Miraldi Utz V, et al. Ophthalmic Genet. 2018 Dec;39(6):671-677. doi: 10.1080/13816810.2018.1533027. Epub 2018 Oct 18. Ophthalmic Genet. 2018. PMID: 30335549 Review.
Of the large number genes that cause IRDs, bi-allelic mutations in RPE65 lead to Leber Congenital Amaurosis type 2 (LCA 2), and can also result in phenotypes described as severe early childhood onset retinal dystrophy (SECORD) and Retinitis pigmentosa 20 (RP2 …
Of the large number genes that cause IRDs, bi-allelic mutations in RPE65 lead to Leber Congenital Amaurosis type 2 (LCA …
Gene therapy for Leber congenital amaurosis: advances and future directions.
Hufnagel RB, Ahmed ZM, Corrêa ZM, Sisk RA. Hufnagel RB, et al. Graefes Arch Clin Exp Ophthalmol. 2012 Aug;250(8):1117-28. doi: 10.1007/s00417-012-2028-2. Epub 2012 May 29. Graefes Arch Clin Exp Ophthalmol. 2012. PMID: 22644094 Free PMC article. Review.
BACKGROUND: Leber congenital amaurosis (LCA) is a congenital retinal dystrophy that results in significant and often severe vision loss at an early age. ...Key terms included "Leber congenital amaurosis", LCA, RPE65, "cone-rod dystrophy", …
BACKGROUND: Leber congenital amaurosis (LCA) is a congenital retinal dystrophy that results in significant and often se …
Gene therapy: perspectives from young adults with Leber's congenital amaurosis.
Napier MP, Selvan K, Hayeems RZ, Shuman C, Chitayat D, Sutherland JE, Day MA, Héon E. Napier MP, et al. Eye (Lond). 2022 Nov;36(11):2088-2093. doi: 10.1038/s41433-021-01763-z. Epub 2021 Sep 16. Eye (Lond). 2022. PMID: 34531550 Free PMC article.
AIMS/PURPOSE: To investigate Leber congenital amaurosis (LCA) patients' expectations, decision-making processes and gene therapy-related concerns. ...DISCUSSION: This study highlights the complex factors involved in gene-therapy-related decision-making and ac …
AIMS/PURPOSE: To investigate Leber congenital amaurosis (LCA) patients' expectations, decision-making processes and gen …
A Mini-Review: Leber Congenital Amaurosis: Identification of Disease-Causing Variants and Personalised Therapies.
Thompson JA, De Roach JN, McLaren TL, Lamey TM. Thompson JA, et al. Adv Exp Med Biol. 2018;1074:265-271. doi: 10.1007/978-3-319-75402-4_32. Adv Exp Med Biol. 2018. PMID: 29721952 Review.
Leber congenital amaurosis (LCA) encompasses a group of severe inherited retinal dystrophies (IRDs) responsible for early childhood blindness. ...At the Australian Inherited Retinal Disease Registry and DNA Bank, we seek to identify genetic variants in indivi …
Leber congenital amaurosis (LCA) encompasses a group of severe inherited retinal dystrophies (IRDs) responsible for ear …
Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy.
Cideciyan AV. Cideciyan AV. Prog Retin Eye Res. 2010 Sep;29(5):398-427. doi: 10.1016/j.preteyeres.2010.04.002. Epub 2010 Apr 24. Prog Retin Eye Res. 2010. PMID: 20399883 Free PMC article. Review.
Leber congenital amaurosis (LCA) is a rare hereditary retinal degeneration caused by mutations in more than a dozen genes. ...Substantial gains in visual function of clinical trial participants provided evidence for physiologically relevant biol …
Leber congenital amaurosis (LCA) is a rare hereditary retinal degeneration caused by mutations in more than a dozen gen …
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial.
Russell SR, Drack AV, Cideciyan AV, Jacobson SG, Leroy BP, Van Cauwenbergh C, Ho AC, Dumitrescu AV, Han IC, Martin M, Pfeifer WL, Sohn EH, Walshire J, Garafalo AV, Krishnan AK, Powers CA, Sumaroka A, Roman AJ, Vanhonsebrouck E, Jones E, Nerinckx F, De Zaeytijd J, Collin RWJ, Hoyng C, Adamson P, Cheetham ME, Schwartz MR, den Hollander W, Asmus F, Platenburg G, Rodman D, Girach A. Russell SR, et al. Nat Med. 2022 May;28(5):1014-1021. doi: 10.1038/s41591-022-01755-w. Epub 2022 Apr 4. Nat Med. 2022. PMID: 35379979 Free PMC article. Clinical Trial.
CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease resulting in childhood blindness. ...The manageable safety profile and improvements reported in this trial support the continuation of sepofarsen development....
CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease resulting in childhood blindness. ... …
136 results