ADAR2 enzymes: efficient site-specific RNA editors with gene
therapy aspirations

J 2022

ADAR2 enzymes: efficient site-specific RNA editors with gene therapy aspirations

HAJJI, Khadija, Jiří SEDMÍK, Anna CHERIAN, Damiano AMORUSO, Liam KEEGAN et. al.

Základní údaje

Originální název

ADAR2 enzymes: efficient site-specific RNA editors with gene therapy aspirations

Autoři

HAJJI, Khadija (788 Tunisko, domácí), Jiří SEDMÍK (203 Česká republika, domácí), Anna CHERIAN (356 Indie, domácí), Damiano AMORUSO (380 Itálie, domácí), Liam KEEGAN (372 Irsko, domácí) a Mary Anne O'CONNELL (372 Irsko, garant, domácí)

Vydání

RNA, Cold Spring Harbor, Cold Spring Harbor Laboratory Press, 2022, 1355-8382

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

10608 Biochemistry and molecular biology

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 4.500

Kód RIV

RIV/00216224:14740/22:00129412

Organizační jednotka

Středoevropský technologický institut

DOI

http://dx.doi.org/10.1261/rna.079266.122

UT WoS

000859478500001

Klíčová slova anglicky

ADAR; ADARB1; dsRNA; recoding RNA editing; neurons

Štítky

rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 3. 4. 2023 10:45, Mgr. Pavla Foltynová, Ph.D.

Anotace

V originále

The adenosine deaminase acting on RNA (ADAR) enzymes are essential for neuronal function and innate immune control. ADAR1 RNA editing prevents aberrant activation of antiviral dsRNA sensors through editing of long, double-stranded RNAs (dsRNAs). In this review, we focus on the ADAR2 proteins involved in the efficient, highly site-specific RNA editing to recode open reading frames first discovered in the GRIA2 transcript encoding the key GLUA2 subunit of AMPA receptors; ADAR1 proteins also edit many of these sites. We summarize the history of ADAR2 protein research and give an up-to-date review of ADAR2 structural studies, human ADARBI (ADAR2) mutants causing severe infant seizures, and mouse disease models. Structural studies on ADARs and their RNA substrates facilitate current efforts to develop ADAR RNA editing gene therapy to edit disease-causing single nucleotide polymorphisms (SNPs). Artificial ADAR guide RNAs are being developed to retarget ADAR RNA editing to new target transcripts in order to correct SNP mutations in them at the RNA level. Site-specific RNA editing has been expanded to recode hundreds of sites in CNS transcripts in Drosophila and cephalopods. In Drosophila and C. elegans, ADAR RNA editing also suppresses responses to self dsRNA.

Návaznosti

GA20-11101S, projekt VaV

Název: Objasnění úlohy RNA-editačního enzymu ADAR1 v nových biologických drahách a určení jeho postranslační regulace.

Investor: Grantová agentura ČR, Elucidating the role of the RNA editing enzyme ADAR1 in novel biological pathways and determine its posttranslational regulation.

GX21-27329X, projekt VaV

Název: ADAR-dependentní RNA editace; Jak imunitní systém a mozek porušují Centrální Dogma.

Investor: Grantová agentura ČR, ADAR RNA editing, how immune systems and brains breach The Central Dogma

956810, interní kód MU

Název: ROles of ePitranscriptomic in diseasES (Akronym: ROPES)

Investor: Evropská unie, ROles of ePitranscriptomic in diseasES, MSCA Marie Skłodowska-Curie Actions (Excellent Science)

Citovat

HAJJI, Khadija, Jiří SEDMÍK, Anna CHERIAN, Damiano AMORUSO, Liam KEEGAN a Mary Anne O'CONNELL. ADAR2 enzymes: efficient site-specific RNA editors with gene therapy aspirations. RNA. Cold Spring Harbor: Cold Spring Harbor Laboratory Press, 2022, roč. 28, č. 10, s. 1281-1297. ISSN 1355-8382. Dostupné z: https://dx.doi.org/10.1261/rna.079266.122.

@article{2252345,
   author = {Hajji, Khadija and Sedmík, Jiří and Cherian, Anna and Amoruso, Damiano and Keegan, Liam and O'Connell, Mary Anne},
   article_location = {Cold Spring Harbor},
   article_number = {10},
   doi = {http://dx.doi.org/10.1261/rna.079266.122},
   keywords = {ADAR; ADARB1; dsRNA; recoding RNA editing; neurons},
   language = {eng},
   issn = {1355-8382},
   journal = {RNA},
   title = {ADAR2 enzymes: efficient site-specific RNA editors with gene therapy aspirations},
   url = {https://rnajournal.cshlp.org/content/28/10/1281},
   volume = {28},
   year = {2022}
}

TY  - JOUR
ID  - 2252345
AU  - Hajji, Khadija - Sedmík, Jiří - Cherian, Anna - Amoruso, Damiano - Keegan, Liam - O'Connell, Mary Anne
PY  - 2022
TI  - ADAR2 enzymes: efficient site-specific RNA editors with gene therapy aspirations
JF  - RNA
VL  - 28
IS  - 10
SP  - 1281-1297
EP  - 1281-1297
PB  - Cold Spring Harbor Laboratory Press
SN  - 13558382
KW  - ADAR
KW  - ADARB1
KW  - dsRNA
KW  - recoding RNA editing
KW  - neurons
UR  - https://rnajournal.cshlp.org/content/28/10/1281
N2  - The adenosine deaminase acting on RNA (ADAR) enzymes are essential for neuronal function and innate immune control. ADAR1 RNA editing prevents aberrant activation of antiviral dsRNA sensors through editing of long, double-stranded RNAs (dsRNAs). In this review, we focus on the ADAR2 proteins involved in the efficient, highly site-specific RNA editing to recode open reading frames first discovered in the GRIA2 transcript encoding the key GLUA2 subunit of AMPA receptors; ADAR1 proteins also edit many of these sites. We summarize the history of ADAR2 protein research and give an up-to-date review of ADAR2 structural studies, human ADARBI (ADAR2) mutants causing severe infant seizures, and mouse disease models. Structural studies on ADARs and their RNA substrates facilitate current efforts to develop ADAR RNA editing gene therapy to edit disease-causing single nucleotide polymorphisms (SNPs). Artificial ADAR guide RNAs are being developed to retarget ADAR RNA editing to new target transcripts in order to correct SNP mutations in them at the RNA level. Site-specific RNA editing has been expanded to recode hundreds of sites in CNS transcripts in Drosophila and cephalopods. In Drosophila and C. elegans, ADAR RNA editing also suppresses responses to self dsRNA.
ER  -

HAJJI, Khadija, Jiří SEDMÍK, Anna CHERIAN, Damiano AMORUSO, Liam KEEGAN a Mary Anne O'CONNELL. ADAR2 enzymes: efficient site-specific RNA editors with gene therapy aspirations. \textit{RNA}. Cold Spring Harbor: Cold Spring Harbor Laboratory Press, 2022, roč.~28, č.~10, s.~1281-1297. ISSN~1355-8382. Dostupné z: https://dx.doi.org/10.1261/rna.079266.122.

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