Extract from the Register of European Patents

EP About this file: EP3177726

EP3177726 - GENOME EDITING FOR THE TREATMENT OF HUNTINGTON'S DISEASE [Right-click to bookmark this link]
Former [2017/24]GENOME EDITING FOR THE TREATMENT OF HUNTINGTON's DISEASE
[2020/36]
StatusNo opposition filed within time limit
Status updated on  03.12.2021
Database last updated on 26.03.2026
FormerThe patent has been granted
Status updated on  25.12.2020
FormerGrant of patent is intended
Status updated on  07.09.2020
FormerExamination is in progress
Status updated on  18.05.2018
FormerRequest for examination was made
Status updated on  12.05.2017
FormerThe international publication has been made
Status updated on  14.02.2017
Most recent event   Tooltip19.12.2025Lapse of the patent in a contracting state
New state(s): TR
published on 21.01.2026  [2026/04]
Applicant(s)For all designated states
Centre Hospitalier Universitaire Vaudois (CHUV)
Rue du Bugnon 21
1011 Lausanne / CH
[2017/24]
Inventor(s)01 / DEGLON, Nicole
Chemin des Biolles 13A
CH-1521 Curtilles / CH
02 / MERIENNE, Nicolas
Rue du Village 42
CH-1081 Montpreveyres / CH
 [2017/24]
Representative(s)KATZAROV S.A.
Geneva Business Center
12 Avenue des Morgines
1213 Petit-Lancy / CH
[N/P]
Former [2021/04]KATZAROV S.A.
European Patent Attorneys
12, Avenue des Morgines
1213 Petit-Lancy / CH
Former [2017/24]KATZAROV S.A.
European Patent Attorneys 19, rue des Epinettes
1227 Genève / CH
Application number, filing date15760097.404.08.2015
[2017/24]
WO2015EP67986
Priority number, dateEP2014017976704.08.2014         Original published format: EP 14179767
[2017/24]
Filing languageEN
Procedural languageEN
PublicationType: A1 Application with search report
No.:WO2016020399
Date:11.02.2016
Language:EN
[2016/06]
Type: A1 Application with search report 
No.:EP3177726
Date:14.06.2017
Language:EN
The application published by WIPO in one of the EPO official languages on 11.02.2016 takes the place of the publication of the European patent application.
[2017/24]
Type: B1 Patent specification 
No.:EP3177726
Date:27.01.2021
Language:EN
[2021/04]
Search report(s)International search report - published on:EP11.02.2016
ClassificationIPC:C12N15/86, C12N9/52, A61K48/00, A61P25/00
[2020/36]
CPC:
A61K48/005 (EP,US); A61K38/1709 (EP,US); A61K38/465 (EP,US);
A61K48/00 (EP,US); C12N15/11 (US); C12N15/86 (EP,US);
C12N9/52 (EP,US); C12N2310/20 (US); C12N2310/3513 (US);
C12N2740/16043 (EP,US); C12N2750/14143 (EP,US); C12N2799/04 (EP,US);
C12N2799/06 (EP,US) (-)
Former IPC [2017/24]C12N15/86, C12N9/52, A61K48/00
Designated contracting statesAL,   AT,   BE,   BG,   CH,   CY,   CZ,   DE,   DK,   EE,   ES,   FI,   FR,   GB,   GR,   HR,   HU,   IE,   IS,   IT,   LI,   LT,   LU,   LV,   MC,   MK,   MT,   NL,   NO,   PL,   PT,   RO,   RS,   SE,   SI,   SK,   SM,   TR [2017/24]
Extension statesBANot yet paid
MENot yet paid
Validation statesMANot yet paid
TitleGerman:GEMONEDITIERUNG ZUR BEHANDLUNG VON MORBUS HUNTINGTON[2020/36]
English:GENOME EDITING FOR THE TREATMENT OF HUNTINGTON'S DISEASE[2020/36]
French:ÉDITION DE GÉNOME POUR LE TRAITEMENT DE LA MALADIE DE HUNTINGTON[2020/36]
Former [2017/24]GENOMÄNDERUNG ZUR BEHANDLUNG VON MORBUS HUNTINGTON
Former [2017/24]GENOME EDITING FOR THE TREATMENT OF HUNTINGTON's DISEASE
Former [2017/24]ÉDITION DU GÉNOME À DES FINS DE TRAITEMENT DE LA MALADIE DE HUNTINGTON
Entry into regional phase14.02.2017National basic fee paid 
14.02.2017Designation fee(s) paid 
14.02.2017Examination fee paid 
Examination procedure14.02.2017Examination requested  [2017/24]
14.02.2017Date on which the examining division has become responsible
23.08.2017Amendment by applicant (claims and/or description)
22.05.2018Despatch of a communication from the examining division (Time limit: M04)
13.09.2018Reply to a communication from the examining division
20.02.2019Despatch of a communication from the examining division (Time limit: M06)
19.08.2019Reply to a communication from the examining division
25.11.2019Despatch of a communication from the examining division (Time limit: M04)
10.03.2020Reply to a communication from the examining division
29.06.2020Despatch of a communication from the examining division (Time limit: M02)
22.07.2020Reply to a communication from the examining division
08.09.2020Communication of intention to grant the patent
16.12.2020Fee for grant paid
16.12.2020Fee for publishing/printing paid
16.12.2020Receipt of the translation of the claim(s)
Opposition(s)28.10.2021No opposition filed within time limit [2022/01]
Fees paidRenewal fee
08.08.2017Renewal fee patent year 03
25.06.2018Renewal fee patent year 04
22.08.2019Renewal fee patent year 05
25.08.2020Renewal fee patent year 06
Opt-out from the exclusive  Tooltip
competence of the Unified
Patent Court
See the Register of the Unified Patent Court for opt-out data
Responsibility for the accuracy, completeness or quality of the data displayed under the link provided lies entirely with the Unified Patent Court.
Lapses during opposition  TooltipHU04.08.2015
AL27.01.2021
AT27.01.2021
CY27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
IT27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
MK27.01.2021
MT27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
TR27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
GB04.08.2021
IE04.08.2021
LU04.08.2021
BE31.08.2021
CH31.08.2021
FR31.08.2021
LI31.08.2021
[2026/04]
Former [2024/41]HU04.08.2015
AL27.01.2021
AT27.01.2021
CY27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
IT27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
MK27.01.2021
MT27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
GB04.08.2021
IE04.08.2021
LU04.08.2021
BE31.08.2021
CH31.08.2021
FR31.08.2021
LI31.08.2021
Former [2024/22]HU04.08.2015
AL27.01.2021
AT27.01.2021
CY27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
IT27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
MK27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
GB04.08.2021
IE04.08.2021
LU04.08.2021
BE31.08.2021
CH31.08.2021
FR31.08.2021
LI31.08.2021
Former [2023/30]HU04.08.2015
AL27.01.2021
AT27.01.2021
CY27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
IT27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
GB04.08.2021
IE04.08.2021
LU04.08.2021
BE31.08.2021
CH31.08.2021
FR31.08.2021
LI31.08.2021
Former [2023/27]HU04.08.2015
AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
IT27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
GB04.08.2021
IE04.08.2021
LU04.08.2021
BE31.08.2021
CH31.08.2021
FR31.08.2021
LI31.08.2021
Former [2022/35]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
IT27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
GB04.08.2021
IE04.08.2021
LU04.08.2021
BE31.08.2021
CH31.08.2021
FR31.08.2021
LI31.08.2021
Former [2022/34]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
IT27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
LU04.08.2021
BE31.08.2021
CH31.08.2021
FR31.08.2021
LI31.08.2021
Former [2022/23]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
IT27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
LU04.08.2021
CH31.08.2021
LI31.08.2021
Former [2022/21]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
PT27.05.2021
CH31.08.2021
LI31.08.2021
Former [2022/18]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
MC27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
PT27.05.2021
Former [2022/13]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SI27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
PT27.05.2021
Former [2022/10]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
PT27.05.2021
Former [2022/08]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
ES27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
Former [2022/07]AL27.01.2021
AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
Former [2021/50]AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RO27.01.2021
RS27.01.2021
SE27.01.2021
SK27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
Former [2021/49]AT27.01.2021
CZ27.01.2021
DK27.01.2021
EE27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RS27.01.2021
SE27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
Former [2021/48]AT27.01.2021
CZ27.01.2021
EE27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RS27.01.2021
SE27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
Former [2021/46]AT27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RS27.01.2021
SE27.01.2021
SM27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
Former [2021/42]AT27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RS27.01.2021
SE27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
IS27.05.2021
PT27.05.2021
Former [2021/39]AT27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
PL27.01.2021
RS27.01.2021
SE27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
PT27.05.2021
Former [2021/38]AT27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
RS27.01.2021
SE27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
PT27.05.2021
Former [2021/37]AT27.01.2021
FI27.01.2021
HR27.01.2021
LT27.01.2021
LV27.01.2021
NL27.01.2021
RS27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
PT27.05.2021
Former [2021/36]FI27.01.2021
HR27.01.2021
LT27.01.2021
NL27.01.2021
BG27.04.2021
NO27.04.2021
GR28.04.2021
PT27.05.2021
Former [2021/35]FI27.01.2021
HR27.01.2021
LT27.01.2021
NL27.01.2021
NO27.04.2021
PT27.05.2021
Former [2021/34]FI27.01.2021
LT27.01.2021
NL27.01.2021
NO27.04.2021
PT27.05.2021
Former [2021/33]FI27.01.2021
LT27.01.2021
NO27.04.2021
PT27.05.2021
Cited inInternational search[A] WO2013130824  (SANGAMO BIOSCIENCES INC et al.)
 [A] WO2014093701  (BROAD INST INC et al.)
 [A]   M. GARRIGA-CANUT ET AL: "Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 109, no. 45, 10 October 2012 (2012-10-10), pages E3136 - E3145, XP055161220, ISSN: 0027-8424, DOI: 10.1073/pnas.1206506109

DOI:   http://dx.doi.org/10.1073/pnas.1206506109
 [A]   CONG LE ET AL: "In Vivo Genome Engineering With AAV Vector Carrying CRISPR-Cas9 System", MOLECULAR THERAPY, vol. 22, no. Suppl. 1, May 2014 (2014-05-01), & 17TH ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-GENE-AND-CELL-THERAPY (ASGCT); WASHINGTON, DC, USA; MAY 21 -24, 2014, pages S214, XP002734380
 [T]   A. FISZER ET AL: "Oligonucleotide-based strategies to combat polyglutamine diseases", NUCLEIC ACIDS RESEARCH, vol. 42, no. 11, 21 May 2014 (2014-05-21), pages 6787 - 6810, XP055161218, ISSN: 0305-1048, DOI: 10.1093/nar/gku385

DOI:   http://dx.doi.org/10.1093/nar/gku385
 [T]   SAMUEL H. STERNBERG ET AL: "DNA interrogation by the CRISPR RNA-guided endonuclease Cas9", NATURE, vol. 507, no. 7490, 29 January 2014 (2014-01-29), pages 62 - 67, XP055161285, ISSN: 0028-0836, DOI: 10.1038/nature13011

DOI:   http://dx.doi.org/10.1038/nature13011
 [T]   MICHELE SIMONATO ET AL: "Progress in gene therapy for neurological disorders", NATURE REVIEWS NEUROLOGY, vol. 9, no. 5, 23 April 2013 (2013-04-23), pages 277 - 291, XP055161287, ISSN: 1759-4758, DOI: 10.1038/nrneurol.2013.56

DOI:   http://dx.doi.org/10.1038/nrneurol.2013.56
 [T]   C. ZUCCATO ET AL: "Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease", PHYSIOLOGICAL REVIEWS, vol. 90, no. 3, 1 July 2010 (2010-07-01), pages 905 - 981, XP055161189, ISSN: 0031-9333, DOI: 10.1152/physrev.00041.2009

DOI:   http://dx.doi.org/10.1152/physrev.00041.2009
Examination  NICOLAS MERIENNE ET AL: "The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes", CELL REPORTS, vol. 20, no. 12, 1 September 2017 (2017-09-01), US, pages 2980 - 2991, XP055548036, ISSN: 2211-1247, DOI: 10.1016/j.celrep.2017.08.075 [DP]

DOI:   http://dx.doi.org/10.1016/j.celrep.2017.08.075
   ZOGHBI H Y ET AL: "Glutamine repeats and neurodegeneration", ANNUAL REVIEW OF NEUROSCIENCE, ANNUAL REVIEWS INC., PALO ALTO, CA, US, vol. 23, 1 January 2000 (2000-01-01), pages 217 - 247, XP002443799, ISSN: 0147-006X, DOI: 10.1146/ANNUREV.NEURO.23.1.217

DOI:   http://dx.doi.org/10.1146/annurev.neuro.23.1.217
by applicantWO2013130824
 WO2014093701
   SMITH; WATERMAN, ADVANCES IN APPLIED MATHEMATICS, vol. 2, 1981, pages 482 - 489
   W.R. PEARSON, METHODS IN ENZYMOLOGY, vol. 183, 1990, pages 63 - 98
   VONSATTEL JP; DIFIGLIA M: "Huntington disease", J NEUROPATH EXP NEUROL, vol. 57, 1998, pages 369 - 384
   ZUCCATO C; VALENZA M; CATTANEO E: "Molecular mechanisms and potential therapeutical targets in Huntington's disease", PHYSIOL REV, vol. 90, no. 3, 2010, pages 905 - 981
   ROSS CA; TABRIZI SJ: "Huntington's disease: from molecular pathogenesis to clinical treatment", LANCET NEURL, vol. 10, no. 1, 2011, pages 83 - 98
   FISZER A; KRZYZOSIAK WJ: "Oligonucleotide-based strategies to combat polyglutamine diseases", NUCLEIC ACIDS RES, vol. 42, no. 11, 2014, pages 6787 - 6810
   DROUET V ET AL.: "Sustained effects of nonallele-specific Huntingtin silencing", ANN NEUROL, vol. 65, no. 3, 2009, pages 276 - 285
   DROUET V ET AL.: "Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells", PLOS ONE, vol. 9, no. 6, 2014, pages E99341
   GARRIGA-CANUT M ET AL.: "Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice", PROC NATL ACAD SCI USA, vol. 109, no. 45, 2012, pages E3136 - 3145
   CONG L ET AL.: "Multiplex genome engineering using CRISPR/Cas systems", SCIENCE, vol. 339, no. 6121, 2013, pages 819 - 823
   JINEK M ET AL.: "RNA-programmed genome editing in human cells", ELIFE, vol. 2, 2013, pages E00471
   STERNBERG SH; REDDING S; JINEK M; GREENE EC; DOUDNA JA: "DNA interrogation by the CRISPR RNA-guided endonuclease Cas9", NATURE, vol. 507, no. 7490, 2014, pages 62 - 67
   MERIENNE N; LE DOUCE J; FAIVRE E; DEGLON N; BONVENTO G: "Efficient gene delivery and selective transduction of astrocytes in the mammalian brain using viral vectors", FRONT CELL NEUROSCI, vol. 7, 2013, pages 106
   SIMONATO M ET AL.: "Progress in gene therapy for neurological disorders", NAT REV NEUROL, vol. 9, no. 5, 2013, pages 277 - 291
   CARTIER N ET AL.: "Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy", SCIENCE, vol. 326, no. 5954, 2009, pages 818 - 823
   PALFI S ET AL.: "Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial", LANCET, vol. 383, no. 9923, 2014, pages 1138 - 1146
   WEINBERG MS; SAMULSKI RJ; MCCOWN TJ: "Adeno-associated virus (AAV) gene therapy for neurological disease", NEUROPHARMACOLOGY, vol. 69, 2013, pages 82 - 88
   NALDINI L ET AL.: "In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector", SCIENCE, vol. 272, no. 5259, 1996, pages 263 - 267
   MALI P ET AL.: "RNA-guided human genome engineering via Cas9", SCIENCE, vol. 339, no. 6121, 2013, pages 823 - 826
   JINEK M ET AL.: "A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity", SCIENCE, vol. 337, no. 6096, 2012, pages 816 - 821
   HIRANO M ET AL.: "Highly efficient retrograde gene transfer into motor neurons by a lentiviral vector pseudotyped with fusion glycoprotein", PLOS ONE, vol. 8, no. 9, 2013, pages E75896
   CASTLE MJ; GERSHENSON ZT; GILES AR; HOLZBAUR EL; WOLFE JH: "Adeno-Associated Virus Serotypes 1, 8, and 9 Share Conserved Mechanisms for Anterograde and Retrograde Axonal Transport", HUM GENE THER, vol. 25, no. 8, 2014, pages 705 - 720
   GRIMM D ET AL.: "In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses", J VIROL, vol. 82, no. 12, 2008, pages 5887 - 5911
   MELO SP ET AL.: "Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant", MOL THER, vol. 22, no. 4, 2014, pages 725 - 733
   DEGLON N ET AL.: "Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease", HUM GENE THER, vol. 11, no. 1, 2000, pages 179 - 190
   HOTTINGER AF; AZZOUZ M; DEGLON N; AEBISCHER P; ZURN AD: "Complete and long-term rescue oflesioned adult motoneurons by lentiviral-mediated expression of glial cell line-derived neurotrophic factor in the facial nucleus", J NEUROSC, vol. 20, no. 15, 2000, pages 5587 - 5593
   DULL T ET AL.: "A third-generation lentivirus vector with a conditional packaging system", J VIROL, vol. 72, no. 11, 1998, pages 8463 - 8471
   LOW K; AEBISCHER P; SCHNEIDER BL: "Direct and retrograde transduction of nigral neurons with AAV6, 8, and 9 and intraneuronal persistence of viral particles", HUM GENE THER, vol. 24, no. 6, 2013, pages 613 - 629
   DRINKUT A; TERESHCHENKO Y; SCHULZ JB; BAHR M; KUGLER S: "Efficient gene therapy for Parkinson's disease using astrocytes as hosts for localized neurotrophic factor delivery", MOL THER, vol. 20, no. 3, 2012, pages 534 - 543
   AURNHAMMER C ET AL.: "Universal real-time PCR for the detection and quantification of adeno-associated virus serotype 2-derived inverted terminal repeat sequences", HUM GENE THER METHODS, vol. 23, no. 1, 2012, pages 18 - 28
   DUJARDIN S ET AL.: "Ectosomes: a new mechanism for non-exosomal secretion of tau protein", PLOS ONE, vol. 9, no. 6, 2014, pages EL00760
   QIU P ET AL.: "Mutation detection using Surveyor nuclease", BIOTECHNIQUES, vol. 36, no. 4, 2004, pages 702 - 707
   RAN FA ET AL.: "Genome engineering using the CRISPR-Cas9 system", NAT PROTOC, vol. 8, no. 11, 2013, pages 2281 - 2308
The EPO accepts no responsibility for the accuracy of data originating from other authorities; in particular, it does not guarantee that it is complete, up to date or fit for specific purposes.