Development and validation of a novel AAV gene therapy for mucopolysaccharidosis type IIIB in large animal

Saved in:
Bibliographic Details
Published inMolecular genetics and metabolism Vol. 141; no. 2; p. 107768
Main Authors Banchi, Elena Gaia, Alonso, Rafael, Jacquot, Sylvie, Fransquin, Kevin, Courtot, Enora, de Saint Denis, Timothee, Ausseil, Jérôme, Piguet, Françoise
Format Journal Article
LanguageEnglish
Published Elsevier Inc 01.02.2024
Online AccessGet full text
ISSN1096-7192
1096-7206
DOI10.1016/j.ymgme.2023.107768

Cover

ArticleNumber 107768
Author Alonso, Rafael
Jacquot, Sylvie
Ausseil, Jérôme
Piguet, Françoise
Fransquin, Kevin
Banchi, Elena Gaia
de Saint Denis, Timothee
Courtot, Enora
Author_xml – sequence: 1
  givenname: Elena Gaia
  surname: Banchi
  fullname: Banchi, Elena Gaia
  organization: Paris Brain Institute, Paris, France
– sequence: 2
  givenname: Rafael
  surname: Alonso
  fullname: Alonso, Rafael
  organization: Paris Brain Institute, Paris, France
– sequence: 3
  givenname: Sylvie
  surname: Jacquot
  fullname: Jacquot, Sylvie
  organization: Paris Brain Institute, Paris, France
– sequence: 4
  givenname: Kevin
  surname: Fransquin
  fullname: Fransquin, Kevin
  organization: Paris Brain Institute, Paris, France
– sequence: 5
  givenname: Enora
  surname: Courtot
  fullname: Courtot, Enora
  organization: Paris Brain Institute, Paris, France
– sequence: 6
  givenname: Timothee
  surname: de Saint Denis
  fullname: de Saint Denis, Timothee
  organization: Paris Brain Institute, Paris, France
– sequence: 7
  givenname: Jérôme
  surname: Ausseil
  fullname: Ausseil, Jérôme
  organization: Toulouse University Hospital, Toulouse, France
– sequence: 8
  givenname: Françoise
  surname: Piguet
  fullname: Piguet, Françoise
  organization: Paris Brain Institute, Paris, France
BookMark eNp9kE1OwzAQhS1UJNrCCdj4Aim20zrOgkUpf5EqsanYWq49bh0ldmSHSrk9KYUtqxnN6L15883QxAcPCN1TsqCE8od6MbSHFhaMsHycFAUXV2hKScmzghE--etpyW7QLKWaEEpX5XKK6mc4QRO6FnyPlTf4pBpnVO-Cx8FihX0Y93i9_sQH8ID7I0TVDdiGiNsvHbrQDElpfVTRmZBcwv3QAa6q6gk7jxsVDzD6ulY1t-jaqibB3W-do93ry27znm0_3qrNepvpUohMC7rUIApeKstyW1pDckb2VGi9VJzmgijDC75iRFlCGRu_sIzTkhf5ikOxz-cov9jqGFKKYGUXx_NxkJTIMy1Zyx9a8kxLXmiNqseLCsZkJwdRJu3AazAugu6lCe5f_TcXmnYQ
ContentType Journal Article
Copyright 2023
Copyright_xml – notice: 2023
DBID AAYXX
CITATION
DOI 10.1016/j.ymgme.2023.107768
DatabaseName CrossRef
DatabaseTitle CrossRef
DeliveryMethod fulltext_linktorsrc
Discipline Anatomy & Physiology
Chemistry
Biology
EISSN 1096-7206
ExternalDocumentID 10_1016_j_ymgme_2023_107768
S1096719223003980
GroupedDBID ---
--K
--M
-~X
.55
.GJ
.~1
0R~
123
1B1
1RT
1~.
1~5
29M
4.4
457
4G.
53G
5RE
5VS
7-5
71M
8P~
AACTN
AAEDT
AAEDW
AAIAV
AAIKJ
AAKOC
AALRI
AAOAW
AAQFI
AAQXK
AAXUO
ABFNM
ABFRF
ABGSF
ABJNI
ABMAC
ABUDA
ABXDB
ABYKQ
ACDAQ
ACGFO
ACGFS
ACRLP
ADBBV
ADEZE
ADFGL
ADMUD
ADUVX
AEBSH
AEFWE
AEHWI
AEKER
AENEX
AFKWA
AFTJW
AFXIZ
AGHFR
AGRDE
AGUBO
AGYEJ
AHPSJ
AIEXJ
AIKHN
AITUG
AJBFU
AJOXV
ALMA_UNASSIGNED_HOLDINGS
AMFUW
AMRAJ
ASPBG
AVWKF
AXJTR
AZFZN
BKOJK
BLXMC
CAG
COF
CS3
DM4
DOVZS
DU5
EBS
EFBJH
EFLBG
EJD
EO8
EO9
EP2
EP3
F5P
FDB
FEDTE
FGOYB
FIRID
FNPLU
FYGXN
G-Q
GBLVA
HVGLF
HZ~
IHE
J1W
K-O
KOM
L7B
LG5
M41
MO0
N9A
O-L
O9-
OAUVE
OZT
P-8
P-9
P2P
PC.
Q38
R2-
RIG
ROL
RPZ
SDF
SDG
SDP
SES
SEW
SPCBC
SSU
SSZ
T5K
X7M
XPP
ZA5
ZGI
ZMT
ZU3
~G-
~KM
AATTM
AAXKI
AAYWO
AAYXX
ABWVN
ACLOT
ACRPL
ACVFH
ADCNI
ADNMO
AEIPS
AEUPX
AFJKZ
AFPUW
AGQPQ
AIGII
AIIUN
AKBMS
AKRWK
AKYEP
ANKPU
APXCP
CITATION
EFKBS
~HD
ID FETCH-LOGICAL-c988-c814ce8769af23f9fd0320b18cc4a61380ad676520af0122594f261967356e7b3
IEDL.DBID .~1
ISSN 1096-7192
IngestDate Wed Oct 01 03:49:38 EDT 2025
Fri Feb 23 02:35:16 EST 2024
IsPeerReviewed true
IsScholarly true
Issue 2
Language English
LinkModel DirectLink
MergedId FETCHMERGED-LOGICAL-c988-c814ce8769af23f9fd0320b18cc4a61380ad676520af0122594f261967356e7b3
ParticipantIDs crossref_primary_10_1016_j_ymgme_2023_107768
elsevier_sciencedirect_doi_10_1016_j_ymgme_2023_107768
ProviderPackageCode CITATION
AAYXX
PublicationCentury 2000
PublicationDate February 2024
2024-02-00
PublicationDateYYYYMMDD 2024-02-01
PublicationDate_xml – month: 02
  year: 2024
  text: February 2024
PublicationDecade 2020
PublicationTitle Molecular genetics and metabolism
PublicationYear 2024
Publisher Elsevier Inc
Publisher_xml – name: Elsevier Inc
SSID ssj0011594
Score 2.4045653
SourceID crossref
elsevier
SourceType Index Database
Publisher
StartPage 107768
Title Development and validation of a novel AAV gene therapy for mucopolysaccharidosis type IIIB in large animal
URI https://dx.doi.org/10.1016/j.ymgme.2023.107768
Volume 141
hasFullText 1
inHoldings 1
isFullTextHit
isPrint
journalDatabaseRights – providerCode: PRVESC
  databaseName: Baden-Württemberg Complete Freedom Collection (Elsevier)
  customDbUrl:
  eissn: 1096-7206
  dateEnd: 99991231
  omitProxy: true
  ssIdentifier: ssj0011594
  issn: 1096-7192
  databaseCode: GBLVA
  dateStart: 20110101
  isFulltext: true
  titleUrlDefault: https://www.sciencedirect.com
  providerName: Elsevier
– providerCode: PRVESC
  databaseName: Elsevier SD Complete Freedom Collection [SCCMFC]
  customDbUrl:
  eissn: 1096-7206
  dateEnd: 99991231
  omitProxy: true
  ssIdentifier: ssj0011594
  issn: 1096-7192
  databaseCode: ACRLP
  dateStart: 19980101
  isFulltext: true
  titleUrlDefault: https://www.sciencedirect.com
  providerName: Elsevier
– providerCode: PRVESC
  databaseName: Elsevier SD Freedom Collection
  customDbUrl:
  eissn: 1096-7206
  dateEnd: 99991231
  omitProxy: true
  ssIdentifier: ssj0011594
  issn: 1096-7192
  databaseCode: .~1
  dateStart: 19980101
  isFulltext: true
  titleUrlDefault: https://www.sciencedirect.com
  providerName: Elsevier
– providerCode: PRVESC
  databaseName: ScienceDirect Freedom Collection Journals
  customDbUrl:
  eissn: 1096-7206
  dateEnd: 99991231
  omitProxy: true
  ssIdentifier: ssj0011594
  issn: 1096-7192
  databaseCode: AIKHN
  dateStart: 19980101
  isFulltext: true
  titleUrlDefault: https://www.sciencedirect.com
  providerName: Elsevier
– providerCode: PRVLSH
  databaseName: Elsevier Journals
  customDbUrl:
  mediaType: online
  eissn: 1096-7206
  dateEnd: 99991231
  omitProxy: true
  ssIdentifier: ssj0011594
  issn: 1096-7192
  databaseCode: AKRWK
  dateStart: 19980101
  isFulltext: true
  providerName: Library Specific Holdings
link http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV07T8MwELYQCMGCeIryqG5ATIQ6TWInY1qBWhAsPNQtchwHpWrSirZDF347d3kgkBADW-LYkXW-3H12vrtj7CKQ2rMVVxZPXG65XsKt2HSN5XEnlUor9EoUO_zwKAYv7t3IG62xfhMLQ7TK2vZXNr201nVLp5ZmZ5ZlnScb0bdEgIIgmjuBT_t2yv6FOn398UXzQMBTFkOkzhb1bjIPlRyvVf6WU67MroMtUlK-1d-80zePc7vLdmqoCGE1mz22Zop9dhAWuE3OV3AJJXmzPBXfZ5u95mqr35RwO2Djb5QgUEUCqFZZVUQJpikoKKb4HMLwFVCPDFTBWCtAIAv5UlP9hNVcaYrMypLpPJsDndjCcDjsQVbAhFjk-N4sV5ND9nx789wfWHVxBUsH-HFo33a1QVMYqLTrpEGaUCX12Pa1dhW6eJ-rREjhdblK6e8bSjGlzZaQjieMjJ0jtl5MC3PMIBae4yotVIzgy1c6IFqY8IxIXdtwKVrsqpFpNKtSaEQNt2wclUsQ0RJE1RK0mGjkHv3QhAiN_F8DT_478JRt451bcbHP2PrifWnOEWos4napS222EQ7vB4-fQ9zSUQ
linkProvider Elsevier
linkToHtml http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1LS-RAEC5kRNyL-FrW9VUH8bRhOpN-JMc4KBMfc3FWvIVOp7NETEZ29DD_3uo8REE8eAv9CKG6UvV191dVACeRMsLXTHss58zjImdeZkfWEywolDaavJKLHb6Zyslffnkv7ldg3MfCOFplZ_tbm95Y665l2Elz-FSWw1uf0LcigEIgmgVRSPv2VS7IJg9gNU6uJtO3ywTy2M3lMo333IQ--VBD81pW_yqXLnMUUItSLuXqZw7qndO52ISNDi1i3H7QFqzYeht24pp2ytUST7HhbzYH49uwdtY_rY_7Km478PCOFYS6zpE0q2zrKOG8QI31nPoxju-QVMliG4-1RMKyWL0YV0JhudDGBWeV-XxRLtAd2mKSJGdY1vjoiOT03rLSj7swuzifjSdeV1_BMxH9Hyb0ubFkDSNdjIIiKnJXTD3zQ2O4Ji8fMp1LJcWI6cJdwJEUC7ffkioQ0qos-AmDel7bX4CZFAHXRuqM8FeoTeSYYVJYWXDfMiX34E8v0_SpzaKR9vSyh7RZgtQtQdouwR7IXu7pB2VIyc5_NfH3dycew_pkdnOdXifTq334QT28pWYfwOD5_4s9JOTxnB11mvUKfNfU_A
openUrl ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=Development+and+validation+of+a+novel+AAV+gene+therapy+for+mucopolysaccharidosis+type+IIIB+in+large+animal&rft.jtitle=Molecular+genetics+and+metabolism&rft.au=Banchi%2C+Elena+Gaia&rft.au=Alonso%2C+Rafael&rft.au=Jacquot%2C+Sylvie&rft.au=Fransquin%2C+Kevin&rft.date=2024-02-01&rft.pub=Elsevier+Inc&rft.issn=1096-7192&rft.eissn=1096-7206&rft.volume=141&rft.issue=2&rft_id=info:doi/10.1016%2Fj.ymgme.2023.107768&rft.externalDocID=S1096719223003980
thumbnail_l http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=1096-7192&client=summon
thumbnail_m http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=1096-7192&client=summon
thumbnail_s http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=1096-7192&client=summon