Molecular Therapies for Inherited Retinal Diseases (Record no. 3007590)

MARC details
000 -LEADER
fixed length control field 05941naaaa2201345uu 4500
001 - CONTROL NUMBER
control field https://directory.doabooks.org/handle/20.500.12854/69174
005 - DATE AND TIME OF LATEST TRANSACTION
control field 20220714185122.0
020 ## - INTERNATIONAL STANDARD BOOK NUMBER
International Standard Book Number books978-3-03943-177-9
020 ## - INTERNATIONAL STANDARD BOOK NUMBER
International Standard Book Number 9783039431762
020 ## - INTERNATIONAL STANDARD BOOK NUMBER
International Standard Book Number 9783039431779
024 7# - OTHER STANDARD IDENTIFIER
Standard number or code 10.3390/books978-3-03943-177-9
Terms of availability doi
041 0# - LANGUAGE CODE
Language code of text/sound track or separate title English
042 ## - AUTHENTICATION CODE
Authentication code dc
072 #7 - SUBJECT CATEGORY CODE
Subject category code GP
Source bicssc
072 #7 - SUBJECT CATEGORY CODE
Subject category code PS
Source bicssc
100 1# - MAIN ENTRY--PERSONAL NAME
Personal name Collin, Rob W.J.
Relator code edt
9 (RLIN) 1607267
245 10 - TITLE STATEMENT
Title Molecular Therapies for Inherited Retinal Diseases
260 ## - PUBLICATION, DISTRIBUTION, ETC. (IMPRINT)
Place of publication, distribution, etc Basel, Switzerland
Name of publisher, distributor, etc MDPI - Multidisciplinary Digital Publishing Institute
Date of publication, distribution, etc 2020
300 ## - PHYSICAL DESCRIPTION
Extent 1 electronic resource (262 p.)
506 0# - RESTRICTIONS ON ACCESS NOTE
Terms governing access Open Access
Source of term star
Standardized terminology for access restriction Unrestricted online access
520 ## - SUMMARY, ETC.
Summary, etc Following the implementation of next-generation sequencing technologies (e.g., exome and genome sequencing) in molecular diagnostics, the majority of genetic defects underlying inherited retinal disease (IRD) can readily be identified. In parallel, opportunities to counteract the molecular consequences of these defects are rapidly emerging, providing hope for personalized medicine. 'Classical' gene augmentation therapy has been under study for several genetic subtypes of IRD and can be considered a safe and sometimes effective therapeutic strategy. The recent market approval of the first retinal gene augmentation therapy product (LuxturnaTM, for individuals with bi-allelic RPE65 mutations) by the FDA has not only demonstrated the potential of this specific approach, but also opened avenues for the development of other strategies. However, every gene-or even every mutation-may need a tailor-made therapeutic approach, in order to obtain the most efficacious strategy with minimal risks associated. In addition to gene augmentation therapy, other subtypes of molecular therapy are currently being designed and/or implemented, including splice modulation, DNA or RNA editing, optogenetics and pharmacological modulation. In addition, the development of proper delivery vectors has gained strong attention, and should not be overlooked when designing and testing a novel therapeutic approach. In this Special Issue, we aim to describe the current state of the art of molecular therapeutics for IRD, and discuss existing and novel therapeutic strategies, from idea to implementation, and from bench to bedside.
540 ## - TERMS GOVERNING USE AND REPRODUCTION NOTE
Terms governing use and reproduction Creative Commons
-- https://creativecommons.org/licenses/by/4.0/
-- cc
-- https://creativecommons.org/licenses/by/4.0/
546 ## - LANGUAGE NOTE
Language note English
650 #7 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name as entry element Research & information: general
Source of heading or term bicssc
9 (RLIN) 928234
650 #7 - SUBJECT ADDED ENTRY--TOPICAL TERM
Topical term or geographic name as entry element Biology, life sciences
Source of heading or term bicssc
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term induced pluripotent stem cell (iPSC)
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Uncontrolled term clustered regularly interspaced short palindromic repeats (CRISPR)
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Uncontrolled term homology-directed repair (HDR)
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Uncontrolled term Enhanced S-Cone Syndrome (ESCS)
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Uncontrolled term NR2E3
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Uncontrolled term AAV
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Uncontrolled term retina
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Uncontrolled term gene therapy
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Uncontrolled term dual AAV
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Uncontrolled term gold nanoparticles
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Uncontrolled term DNA-wrapped gold nanoparticles
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Uncontrolled term ARPE-19 cells
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Uncontrolled term retinal pigment epithelium
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Uncontrolled term clathrin-coated vesicles
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Uncontrolled term endosomal trafficking
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Uncontrolled term retinitis pigmentosa
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Uncontrolled term autosomal dominant
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Uncontrolled term G56R
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Uncontrolled term putative dominant negative effect
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Uncontrolled term gapmer antisense oligonucleotides
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Uncontrolled term allele-specific knockdown
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Uncontrolled term Leber congenital amaurosis and allied retinal ciliopathies
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Uncontrolled term CEP290
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Uncontrolled term Flanders founder c.4723A &gt
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Uncontrolled term T nonsense mutation
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Uncontrolled term Cilia elongation
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Uncontrolled term spontaneous nonsense correction
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Uncontrolled term AON-mediated exon skipping
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Uncontrolled term microRNA
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Uncontrolled term photoreceptors
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Uncontrolled term rods
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Uncontrolled term cones
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Uncontrolled term bipolar cells
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Uncontrolled term Müller glia
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Uncontrolled term retinal inherited disorders
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Uncontrolled term retinal degeneration
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Uncontrolled term antisense oligonucleotides
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Uncontrolled term Stargardt disease
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Uncontrolled term inherited retinal diseases
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term splicing modulation
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Uncontrolled term RNA therapy
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term ABCA4
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term iPSC-derived photoreceptor precursor cells
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Uncontrolled term cyclic GMP
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Uncontrolled term apoptosis
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Uncontrolled term necrosis
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Uncontrolled term drug delivery systems
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Uncontrolled term translational medicine
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Uncontrolled term Usher syndrome
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Uncontrolled term Leber congenital amaurosis
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Uncontrolled term RPE65
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Uncontrolled term nonprofit
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Uncontrolled term patient registry
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term translational
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Uncontrolled term protein trafficking
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Uncontrolled term protein folding
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Uncontrolled term protein degradation
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Uncontrolled term chaperones
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Uncontrolled term chaperonins
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Uncontrolled term heat shock response
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Uncontrolled term unfolded protein response
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term autophagy
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term therapy
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term IRD
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Uncontrolled term DNA therapies
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Uncontrolled term RNA therapies
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Uncontrolled term compound therapies
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Uncontrolled term clinical trials
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Uncontrolled term Retinitis Pigmentosa GTPase Regulator
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Uncontrolled term adeno-associated viral
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Uncontrolled term Retinitis Pigmentosa (RP)
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Uncontrolled term choroideremia
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Uncontrolled term REP1
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term inherited retinal disease
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Uncontrolled term treatment
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term apical polarity
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term crumbs complex
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term fetal retina
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Uncontrolled term PAR complex
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term retinal organoids
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term retinogenesis
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term gene augmentation
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term adeno-associated virus (AAV)
653 ## - INDEX TERM--UNCONTROLLED
Uncontrolled term n/a
700 1# - ADDED ENTRY--PERSONAL NAME
Personal name Garanto, Alejandro
Relator code edt
9 (RLIN) 1563091
700 1# - ADDED ENTRY--PERSONAL NAME
Personal name Collin, Rob W.J.
Relator code oth
9 (RLIN) 1607267
700 1# - ADDED ENTRY--PERSONAL NAME
Personal name Garanto, Alejandro
Relator code oth
9 (RLIN) 1563091
856 40 - ELECTRONIC LOCATION AND ACCESS
Host name www.oapen.org
Uniform Resource Identifier <a href="https://mdpi.com/books/pdfview/book/2946">https://mdpi.com/books/pdfview/book/2946</a>
-- 0
Public note DOAB: download the publication
856 40 - ELECTRONIC LOCATION AND ACCESS
Host name www.oapen.org
Uniform Resource Identifier <a href="https://directory.doabooks.org/handle/20.500.12854/69174">https://directory.doabooks.org/handle/20.500.12854/69174</a>
-- 0
Public note DOAB: description of the publication
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