schliessen

Filtern

 

Bibliotheken

Refining strategies to translate genome editing to the clinic

Recent progress in developing programmable nucleases, such as zinc-finger nucleases, transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR)-Cas nucleases, have paved the way for gene editing to enter clinical practice. This tran... Full description

Journal Title: Nature Medicine 2017-04-03, Vol.23 (4), p.415-423
Main Author: Cornu, Tatjana I
Other Authors: Mussolino, Claudio , Cathomen, Toni
Format: Electronic Article Electronic Article
Language: English
Subjects:
Publisher: United States: Nature Publishing Group
ID: ISSN: 1078-8956
Link: https://www.ncbi.nlm.nih.gov/pubmed/28388605
Zum Text:
SendSend as email Add to Book BagAdd to Book Bag
Staff View
recordid: cdi_proquest_miscellaneous_1891852529
title: Refining strategies to translate genome editing to the clinic
format: Article
creator:
  • Cornu, Tatjana I
  • Mussolino, Claudio
  • Cathomen, Toni
subjects:
  • Clinical Trials as Topic
  • CRISPR-Cas Systems
  • Deoxyribonucleases
  • Dependovirus
  • Disease
  • Gene Editing - legislation & jurisprudence
  • Gene Editing - methods
  • Gene therapy
  • Genetic engineering
  • Genetic research
  • Genetic Vectors
  • Genetics
  • Genomes
  • Hemoglobinopathies - therapy
  • Hemophilia A - therapy
  • HIV Infections - therapy
  • Humans
  • Immunologic Deficiency Syndromes - therapy
  • Innovations
  • Leukemia - therapy
  • Muscular Dystrophies - therapy
  • Neoplasms - therapy
  • Nucleases
  • Properties
  • Transcription Activator-Like Effector Nucleases
ispartof: Nature Medicine, 2017-04-03, Vol.23 (4), p.415-423
description: Recent progress in developing programmable nucleases, such as zinc-finger nucleases, transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR)-Cas nucleases, have paved the way for gene editing to enter clinical practice. This translation is a result of combining high nuclease activity with high specificity and successfully applying this technology in various preclinical disease models, including infectious disease, primary immunodeficiencies, hemoglobinopathies, hemophilia and muscular dystrophy. Several clinical gene-editing trials, both ex vivo and in vivo, have been initiated in the past 2 years, including studies that aim to knockout genes as well as to add therapeutic transgenes. Here we discuss the advances made in the gene-editing field in recent years, and specify priorities that need to be addressed to expand therapeutic genome editing to further disease entities.
language: eng
source:
identifier: ISSN: 1078-8956
fulltext: no_fulltext
issn:
  • 1078-8956
  • 1546-170X
url: Link


@attributes
NO1
SEARCH_ENGINEprimo_central_multiple_fe
SEARCH_ENGINE_TYPEPrimo Central Search Engine
RANK2.7986755
LOCALfalse
PrimoNMBib
record
control
sourceidgale_opena
recordidTN_cdi_proquest_miscellaneous_1891852529
sourceformatXML
sourcesystemPC
galeidA490684509
sourcerecordidA490684509
originalsourceidFETCH-LOGICAL-c586t-3552ee712697f35f50a42b06833693a8628305b00702e7107481999f2dbf92900
addsrcrecordideNqNkt9r1TAUx4sobk7xP5CC4I-HXk-Spk0efBhD52AwmD_wLaTpaW9Gm9w1Kbj_3tSr2-4cYvKQX5_zPT9ysuw5gRUBJt65cVUywh5k-4SXVUFq-P4w7aEWhZC82suehHABAAy4fJztUcGEqIDvZ-_PsbPOuj4PcdIRe4shjz5PBxeGdJH36PyIObY2LtjytsbcDMnKPM0edXoI-Oz3epB9_fjhy9Gn4vTs-OTo8LQwXFSxYJxTxJrQStYd4x0HXdIGKsFYJZkWVYoHeANQA00Y1KUgUsqOtk0nqQQ4yI63un6DTtsJ1Wayo56ulNdWtQ6jClf9qBVr2tpga1ipZduJpkXaogBoKSGGapmU3myVNpO_nDFENdpgcBi0Qz8HRYQkglNO_wcVXJZpLPG9vINe-HlyqSS_KMpYXVU3VK8HVNZ1PlXZLKLqsJSpHCWHxe3qHirNFkdrvEv_le53DF7cY6BuA293gCQS8Ufs9RyCOvl8viv2L_bs2y776ha7Rj3EdfDDHK13YRd8vQXN5EOYsLv-PgJq6V_lRrX0700um7kZsb3m_jRsAoo7UsZGvXhMSdvhL8Gf6Vnwcw
sourcetypeOpen Access Repository
isCDItrue
recordtypearticle
pqid1885233766
display
typearticle
titleRefining strategies to translate genome editing to the clinic
creatorCornu, Tatjana I ; Mussolino, Claudio ; Cathomen, Toni
creatorcontribCornu, Tatjana I ; Mussolino, Claudio ; Cathomen, Toni
descriptionRecent progress in developing programmable nucleases, such as zinc-finger nucleases, transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR)-Cas nucleases, have paved the way for gene editing to enter clinical practice. This translation is a result of combining high nuclease activity with high specificity and successfully applying this technology in various preclinical disease models, including infectious disease, primary immunodeficiencies, hemoglobinopathies, hemophilia and muscular dystrophy. Several clinical gene-editing trials, both ex vivo and in vivo, have been initiated in the past 2 years, including studies that aim to knockout genes as well as to add therapeutic transgenes. Here we discuss the advances made in the gene-editing field in recent years, and specify priorities that need to be addressed to expand therapeutic genome editing to further disease entities.
identifier
0ISSN: 1078-8956
1EISSN: 1546-170X
2DOI: 10.1038/nm.4313
3PMID: 28388605
languageeng
publisherUnited States: Nature Publishing Group
subjectClinical Trials as Topic ; CRISPR-Cas Systems ; Deoxyribonucleases ; Dependovirus ; Disease ; Gene Editing - legislation & jurisprudence ; Gene Editing - methods ; Gene therapy ; Genetic engineering ; Genetic research ; Genetic Vectors ; Genetics ; Genomes ; Hemoglobinopathies - therapy ; Hemophilia A - therapy ; HIV Infections - therapy ; Humans ; Immunologic Deficiency Syndromes - therapy ; Innovations ; Leukemia - therapy ; Muscular Dystrophies - therapy ; Neoplasms - therapy ; Nucleases ; Properties ; Transcription Activator-Like Effector Nucleases
ispartofNature Medicine, 2017-04-03, Vol.23 (4), p.415-423
rights
0COPYRIGHT 2017 Nature Publishing Group
1Copyright Nature Publishing Group Apr 2017
lds50peer_reviewed
citedbyFETCH-LOGICAL-c586t-3552ee712697f35f50a42b06833693a8628305b00702e7107481999f2dbf92900
citesFETCH-LOGICAL-c586t-3552ee712697f35f50a42b06833693a8628305b00702e7107481999f2dbf92900
orcidid0000-0002-7757-4630
links
openurl$$Topenurl_article
thumbnail$$Usyndetics_thumb_exl
backlink$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/28388605$$D View this record in MEDLINE/PubMed
search
creatorcontrib
0Cornu, Tatjana I
1Mussolino, Claudio
2Cathomen, Toni
title
0Refining strategies to translate genome editing to the clinic
1Nature Medicine
addtitleNat Med
descriptionRecent progress in developing programmable nucleases, such as zinc-finger nucleases, transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR)-Cas nucleases, have paved the way for gene editing to enter clinical practice. This translation is a result of combining high nuclease activity with high specificity and successfully applying this technology in various preclinical disease models, including infectious disease, primary immunodeficiencies, hemoglobinopathies, hemophilia and muscular dystrophy. Several clinical gene-editing trials, both ex vivo and in vivo, have been initiated in the past 2 years, including studies that aim to knockout genes as well as to add therapeutic transgenes. Here we discuss the advances made in the gene-editing field in recent years, and specify priorities that need to be addressed to expand therapeutic genome editing to further disease entities.
subject
0Clinical Trials as Topic
1CRISPR-Cas Systems
2Deoxyribonucleases
3Dependovirus
4Disease
5Gene Editing - legislation & jurisprudence
6Gene Editing - methods
7Gene therapy
8Genetic engineering
9Genetic research
10Genetic Vectors
11Genetics
12Genomes
13Hemoglobinopathies - therapy
14Hemophilia A - therapy
15HIV Infections - therapy
16Humans
17Immunologic Deficiency Syndromes - therapy
18Innovations
19Leukemia - therapy
20Muscular Dystrophies - therapy
21Neoplasms - therapy
22Nucleases
23Properties
24Transcription Activator-Like Effector Nucleases
issn
01078-8956
11546-170X
fulltextfalse
rsrctypearticle
creationdate2017
recordtypearticle
recordideNqNkt9r1TAUx4sobk7xP5CC4I-HXk-Spk0efBhD52AwmD_wLaTpaW9Gm9w1Kbj_3tSr2-4cYvKQX5_zPT9ysuw5gRUBJt65cVUywh5k-4SXVUFq-P4w7aEWhZC82suehHABAAy4fJztUcGEqIDvZ-_PsbPOuj4PcdIRe4shjz5PBxeGdJH36PyIObY2LtjytsbcDMnKPM0edXoI-Oz3epB9_fjhy9Gn4vTs-OTo8LQwXFSxYJxTxJrQStYd4x0HXdIGKsFYJZkWVYoHeANQA00Y1KUgUsqOtk0nqQQ4yI63un6DTtsJ1Wayo56ulNdWtQ6jClf9qBVr2tpga1ipZduJpkXaogBoKSGGapmU3myVNpO_nDFENdpgcBi0Qz8HRYQkglNO_wcVXJZpLPG9vINe-HlyqSS_KMpYXVU3VK8HVNZ1PlXZLKLqsJSpHCWHxe3qHirNFkdrvEv_le53DF7cY6BuA293gCQS8Ufs9RyCOvl8viv2L_bs2y776ha7Rj3EdfDDHK13YRd8vQXN5EOYsLv-PgJq6V_lRrX0700um7kZsb3m_jRsAoo7UsZGvXhMSdvhL8Gf6Vnwcw
startdate20170403
enddate20170403
creator
0Cornu, Tatjana I
1Mussolino, Claudio
2Cathomen, Toni
generalNature Publishing Group
scope
0CGR
1CUY
2CVF
3ECM
4EIF
5NPM
6AAYXX
7CITATION
8IOF
93V.
107QG
117QL
127QP
137QR
147T5
157TK
167TM
177TO
187U7
197U9
207X7
217XB
2288A
2388E
2488I
258AO
268FD
278FE
288FH
298FI
308FJ
318FK
328G5
33ABUWG
34AZQEC
35BBNVY
36BENPR
37BHPHI
38C1K
39DWQXO
40FR3
41FYUFA
42GHDGH
43GNUQQ
44GUQSH
45H94
46HCIFZ
47K9.
48LK8
49M0S
50M1P
51M2O
52M2P
53M7N
54M7P
55MBDVC
56P64
57PADUT
58PQEST
59PQQKQ
60PQUKI
61PRINS
62Q9U
63RC3
647X8
65CLFQK
orcididhttps://orcid.org/0000-0002-7757-4630
sort
creationdate20170403
titleRefining strategies to translate genome editing to the clinic
authorCornu, Tatjana I ; Mussolino, Claudio ; Cathomen, Toni
facets
frbrtype5
frbrgroupidcdi_FETCH-LOGICAL-c586t-3552ee712697f35f50a42b06833693a8628305b00702e7107481999f2dbf92900
rsrctypearticles
prefilterarticles
languageeng
creationdate2017
topic
0Clinical Trials as Topic
1CRISPR-Cas Systems
2Deoxyribonucleases
3Dependovirus
4Disease
5Gene Editing - legislation & jurisprudence
6Gene Editing - methods
7Gene therapy
8Genetic engineering
9Genetic research
10Genetic Vectors
11Genetics
12Genomes
13Hemoglobinopathies - therapy
14Hemophilia A - therapy
15HIV Infections - therapy
16Humans
17Immunologic Deficiency Syndromes - therapy
18Innovations
19Leukemia - therapy
20Muscular Dystrophies - therapy
21Neoplasms - therapy
22Nucleases
23Properties
24Transcription Activator-Like Effector Nucleases
toplevelpeer_reviewed
creatorcontrib
0Cornu, Tatjana I
1Mussolino, Claudio
2Cathomen, Toni
collection
0Medline
1MEDLINE
2MEDLINE (Ovid)
3MEDLINE
4MEDLINE
5PubMed
6CrossRef
7Gale General OneFile
8ProQuest Central (Corporate)
9Animal Behavior Abstracts
10Bacteriology Abstracts (Microbiology B)
11Calcium & Calcified Tissue Abstracts
12Chemoreception Abstracts
13Immunology Abstracts
14Neurosciences Abstracts
15Nucleic Acids Abstracts
16Oncogenes and Growth Factors Abstracts
17Toxicology Abstracts
18Virology and AIDS Abstracts
19Health & Medical Collection
20ProQuest Central (purchase pre-March 2016)
21Biology Database (Alumni Edition)
22Medical Database (Alumni Edition)
23Science Database (Alumni Edition)
24ProQuest Pharma Collection
25Technology Research Database
26ProQuest SciTech Collection
27ProQuest Natural Science Collection
28Hospital Premium Collection
29Hospital Premium Collection (Alumni Edition)
30ProQuest Central (Alumni) (purchase pre-March 2016)
31Research Library (Alumni Edition)
32ProQuest Central (Alumni Edition)
33ProQuest Central Essentials
34Biological Science Collection
35ProQuest Central
36Natural Science Collection
37Environmental Sciences and Pollution Management
38ProQuest Central Korea
39Engineering Research Database
40Health Research Premium Collection
41Health Research Premium Collection (Alumni)
42ProQuest Central Student
43Research Library Prep
44AIDS and Cancer Research Abstracts
45SciTech Premium Collection
46ProQuest Health & Medical Complete (Alumni)
47ProQuest Biological Science Collection
48Health & Medical Collection (Alumni Edition)
49Medical Database
50Research Library
51Science Database
52Algology Mycology and Protozoology Abstracts (Microbiology C)
53Biological Science Database
54Research Library (Corporate)
55Biotechnology and BioEngineering Abstracts
56Research Library China
57ProQuest One Academic Eastern Edition
58ProQuest One Academic
59ProQuest One Academic UKI Edition
60ProQuest Central China
61ProQuest Central Basic
62Genetics Abstracts
63MEDLINE - Academic
64OpenAIRE
jtitleNature Medicine
delivery
delcategoryRemote Search Resource
fulltextno_fulltext
addata
au
0Cornu, Tatjana I
1Mussolino, Claudio
2Cathomen, Toni
formatjournal
genrearticle
ristypeJOUR
atitleRefining strategies to translate genome editing to the clinic
jtitleNature Medicine
addtitleNat Med
date2017-04-03
risdate2017
volume23
issue4
spage415
epage423
pages415-423
issn1078-8956
eissn1546-170X
abstractRecent progress in developing programmable nucleases, such as zinc-finger nucleases, transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR)-Cas nucleases, have paved the way for gene editing to enter clinical practice. This translation is a result of combining high nuclease activity with high specificity and successfully applying this technology in various preclinical disease models, including infectious disease, primary immunodeficiencies, hemoglobinopathies, hemophilia and muscular dystrophy. Several clinical gene-editing trials, both ex vivo and in vivo, have been initiated in the past 2 years, including studies that aim to knockout genes as well as to add therapeutic transgenes. Here we discuss the advances made in the gene-editing field in recent years, and specify priorities that need to be addressed to expand therapeutic genome editing to further disease entities.
copUnited States
pubNature Publishing Group
pmid28388605
doi10.1038/nm.4313
tpages9
orcididhttps://orcid.org/0000-0002-7757-4630