Molecular Therapies for Inherited Retinal Diseases
| Weitere Verfasser: |
Collin, Rob W.J.
, [HerausgeberIn]
Garanto, Alejandro , [HerausgeberIn] |
|---|---|
| Umfang/Format: |
1 online resource (262 pages). |
| Schlagworte: | |
| Online Zugang: |
DOAB: download the publication DOAB: description of the publication |
| LEADER | 04720namaa2201489ui 4500 | ||
|---|---|---|---|
| 001 | 003028466 | ||
| 005 | 20221228154510.0 | ||
| 003 | DE-2553 | ||
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| 008 | 20210501s2020 xx |||||o ||| 0|eng d | ||
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| 024 | 7 | |a 10.3390/books978-3-03943-177-9 |c doi | |
| 041 | 0 | |a eng | |
| 042 | |a dc | ||
| 072 | 7 | |a GP |2 bicssc | |
| 072 | 7 | |a PS |2 bicssc | |
| 100 | 1 | |a Collin, Rob W.J. |e editor | |
| 264 | |b MDPI - Multidisciplinary Digital Publishing Institute, |c 2020. | ||
| 700 | 1 | |a Garanto, Alejandro |e editor | |
| 700 | 1 | |a Collin, Rob W.J. |e other | |
| 700 | 1 | |a Garanto, Alejandro |e other | |
| 245 | 1 | 0 | |a Molecular Therapies for Inherited Retinal Diseases |
| 300 | |a 1 online resource (262 pages). | ||
| 336 | |a text |b txt |2 rdacontent | ||
| 337 | |a computer |b c |2 rdamedia | ||
| 338 | |a online resource |b cr |2 rdacarrier | ||
| 506 | 0 | |a Open Access |2 star |f Unrestricted online access | |
| 540 | |a Creative Commons |f https://creativecommons.org/licenses/by/4.0/ |2 cc |4 https://creativecommons.org/licenses/by/4.0/ | ||
| 546 | |a English | ||
| 650 | 7 | |a Research & information: general |2 bicssc | |
| 650 | 7 | |a Biology, life sciences |2 bicssc | |
| 653 | |a induced pluripotent stem cell (iPSC) | ||
| 653 | |a clustered regularly interspaced short palindromic repeats (CRISPR) | ||
| 653 | |a homology-directed repair (HDR) | ||
| 653 | |a Enhanced S-Cone Syndrome (ESCS) | ||
| 653 | |a NR2E3 | ||
| 653 | |a AAV | ||
| 653 | |a retina | ||
| 653 | |a gene therapy | ||
| 653 | |a dual AAV | ||
| 653 | |a gold nanoparticles | ||
| 653 | |a DNA-wrapped gold nanoparticles | ||
| 653 | |a ARPE-19 cells | ||
| 653 | |a retinal pigment epithelium | ||
| 653 | |a clathrin-coated vesicles | ||
| 653 | |a endosomal trafficking | ||
| 653 | |a retinitis pigmentosa | ||
| 653 | |a autosomal dominant | ||
| 653 | |a G56R | ||
| 653 | |a putative dominant negative effect | ||
| 653 | |a gapmer antisense oligonucleotides | ||
| 653 | |a allele-specific knockdown | ||
| 653 | |a Leber congenital amaurosis and allied retinal ciliopathies | ||
| 653 | |a CEP290 | ||
| 653 | |a Flanders founder c.4723A > | ||
| 653 | |a T nonsense mutation | ||
| 653 | |a Cilia elongation | ||
| 653 | |a spontaneous nonsense correction | ||
| 653 | |a AON-mediated exon skipping | ||
| 653 | |a microRNA | ||
| 653 | |a photoreceptors | ||
| 653 | |a rods | ||
| 653 | |a cones | ||
| 653 | |a bipolar cells | ||
| 653 | |a Müller glia | ||
| 653 | |a retinal inherited disorders | ||
| 653 | |a retinal degeneration | ||
| 653 | |a antisense oligonucleotides | ||
| 653 | |a Stargardt disease | ||
| 653 | |a inherited retinal diseases | ||
| 653 | |a splicing modulation | ||
| 653 | |a RNA therapy | ||
| 653 | |a ABCA4 | ||
| 653 | |a iPSC-derived photoreceptor precursor cells | ||
| 653 | |a cyclic GMP | ||
| 653 | |a apoptosis | ||
| 653 | |a necrosis | ||
| 653 | |a drug delivery systems | ||
| 653 | |a translational medicine | ||
| 653 | |a Usher syndrome | ||
| 653 | |a Leber congenital amaurosis | ||
| 653 | |a RPE65 | ||
| 653 | |a nonprofit | ||
| 653 | |a patient registry | ||
| 653 | |a translational | ||
| 653 | |a protein trafficking | ||
| 653 | |a protein folding | ||
| 653 | |a protein degradation | ||
| 653 | |a chaperones | ||
| 653 | |a chaperonins | ||
| 653 | |a heat shock response | ||
| 653 | |a unfolded protein response | ||
| 653 | |a autophagy | ||
| 653 | |a therapy | ||
| 653 | |a IRD | ||
| 653 | |a DNA therapies | ||
| 653 | |a RNA therapies | ||
| 653 | |a compound therapies | ||
| 653 | |a clinical trials | ||
| 653 | |a Retinitis Pigmentosa GTPase Regulator | ||
| 653 | |a adeno-associated viral | ||
| 653 | |a Retinitis Pigmentosa (RP) | ||
| 653 | |a choroideremia | ||
| 653 | |a REP1 | ||
| 653 | |a inherited retinal disease | ||
| 653 | |a treatment | ||
| 653 | |a apical polarity | ||
| 653 | |a crumbs complex | ||
| 653 | |a fetal retina | ||
| 653 | |a PAR complex | ||
| 653 | |a retinal organoids | ||
| 653 | |a retinogenesis | ||
| 653 | |a gene augmentation | ||
| 653 | |a adeno-associated virus (AAV) | ||
| 653 | |a n/a | ||
| 856 | 4 | 0 | |a www.oapen.org |u https://mdpi.com/books/pdfview/book/2946 |7 0 |z DOAB: download the publication |
| 856 | 4 | 0 | |a www.oapen.org |u https://directory.doabooks.org/handle/20.500.12854/69174 |7 0 |z DOAB: description of the publication |
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