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AAV Serotypes and Their Suitability for Retinal Gene Therapy
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- المؤلفون: Ash, John; Pierce, Eric; Anderson, Robert; Bowes Rickman, Catherine; Hollyfield, Joe G; Grimm, Christian; Grimm, Christian; Ash, J ( John ); Pierce, E ( Eric ); Anderson, R ( Robert ); Bowes Rickman, C ( Catherine ); Hollyfield, J G ( Joe G ); Grimm, C ( Christian ); Ebner, Lynn J A; Ebner, Lynn J A
- المصدر:
Ebner, Lynn J A; Grimm, Christian (2023). AAV Serotypes and Their Suitability for Retinal Gene Therapy. In: Ash, John; Pierce, Eric; Anderson, Robert; Bowes Rickman, Catherine; Hollyfield, Joe G; Grimm, Christian. Retinal Degenerative Diseases XIX : Mechanisms and Experimental Therapy. Cham: Springer, 131-134.
- نوع التسجيلة:
Electronic Resource
- الدخول الالكتروني :
https://www.zora.uzh.ch/id/eprint/234799/1/Ebner_Grimm_2023.pdf
https://www.zora.uzh.ch/id/eprint/234799/
https://www.zora.uzh.ch/id/eprint/234799
Online ISBN: 9783031276811
10.1007/978-3-031-27681-1_20
- معلومة اضافية
- Publisher Information:
Springer 2023-07-14
- نبذة مختصرة :
Throughout the last 25 years, exceptional progress in retinal gene therapy was achieved. The major breakthrough was realized in 2017 when the FDA approved the adeno-associated virus (AAV)-based gene therapy for treatment of the monogenetic disorder Leber congenital amaurosis type 2 (LCA2). Since then, many therapies for inherited retinal diseases (IRD) reached phase I/II clinical trials, targeting diseases like achromatopsia, choroideremia, retinitis pigmentosa, Stargardt disease, and many more (reviewed in (Trapani and Auricchio, Trends Mol Med 24:669–681, 2018)). Advanced vector and capsid design technologies as well as improved gene transfer and gene editing methods may lead to refined therapies for various eye diseases. Many research departments worldwide focus on optimizing transgene expression by designing novel AAV serotypes. Besides serotype tropism, the method of injection (intravitreal, subretinal, or suprachoroidal) (Han et al., Hum Gene Ther 31:1288–1299, 2020) defines the efficiency outcome along with the use of tissue-specific promotors which play a critical role for cell targeting.
- الموضوع:
- Availability:
Open access content. Open access content
info:eu-repo/semantics/closedAccess
info:eu-repo/semantics/closedAccess
- Note:
application/pdf
English
English
- Other Numbers:
CHUZH oai:www.zora.uzh.ch:234799
info:doi/10.5167/uzh-234799
info:doi/10.1007/978-3-031-27681-1_20
info:pmid/37440025
urn:isbn:9783031276804
urn:issn:0065-2598
1398327346
- Contributing Source:
HAUPTBIBLIOTHEK UNIV OF ZURICH
From OAIster®, provided by the OCLC Cooperative.
- الرقم المعرف:
edsoai.on1398327346
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