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Part 1:  1  2  3  4  5  6          Part 2:  A  B  C  D  E 
International application No. Applicant's or agent's file reference
PCT/US2020/016542 33057/53576/PC
International filing date (day/month/year) (Earliest) Priority Date (day/month/year)
04 February 2020 04 February 2019
FOR FURTHER ACTION: See Form PCT/ISA/220 as well as, where applicable, item 5 below.
This international search report has been prepared by this International Searching Authority and is transmitted to the applicant according to Article 18. A copy is being transmitted to the International Bureau.
It is also accompanied by a copy of each prior art document cited in this report.
1. Basis of the report
a. With regard to the language, the international search was carried out on the basis of:
the international application in the language in which it was filed.
a translation of the international application into                                          which is the language of a translation furnished for the purposes of international search (Rules 12.3(a) and 23.1(b)).
This international search report has been established taking into account the rectification of an obvious mistake authorized by or notified to this Authority under Rule 91 (Rule 43.6bis(a)).
1. With regard to any nucleotide and/or amino acid sequence disclosed in the international application, the international search was carried out on the basis of a sequence listing:
a. forming part of the international application as filed:
in the form of an Annex C/ST.25 text file.
on paper or in the form of an image file.
b. furnished together with the international application under PCT Rule 13ter.1(a) for the purposes of international search only in the form of an Annex C/ST.25 text file.
c. furnished subsequent to the international filing date for the purposes of international search only:
in the form of an Annex C/ST.25 text file (Rule 13ter.1(a)).
on paper or in the form of an image file (Rule 13ter.1(b) and Administrative Instructions, Section 713).
In addition, in the case that more than one version or copy of a sequence listing has been filed or furnished, the required statements that the information in the subsequent or additional copies is identical to that forming part of the application as filed or does not go beyond the application as filed, as appropriate, were furnished.
Additional comments:
2. Certain claims were found unsearchable
3. Unity of invention is lacking
4. Title of the invention
The text is approved as submitted by the applicant.
The text has been established by this Authority to read as follows:
5. Abstract
The text is approved as submitted by the applicant.
The text has been established, according to Rule 38.2, by this Authority as it appears in Box No. IV. The applicant may, within one month from the date of mailing of this international search report, submit comments to this Authority.
6. Drawings
The figure of the drawings to be published with the abstract is Figure No. ____________
as suggested by the applicant.
as selected by this Authority, because the applicant failed to suggest a figure.
as selected by this Authority, because this figure better characterizes the invention.
none of the figures is to be published with the abstract.


     C12N 15/86 (2006.01)i; C07K 14/47 (2006.01)i; C07K 14/435 (2006.01)i
According to International Patent Classification (IPC) or to both national classification and IPC


Minimum documentation searched (classification system followed by classification symbols):
     C12N; C07K
Documentation searched other than minimum documentation to the extent that such documents are included in the fields searched:
Electronic data base consulted during the international search (name of data base and, where practicable, search terms used):


Category* Citation of document, with indication, where appropriate, of the relevant passages Relevant to claim No.
WO 2016100575 A1 (UNIV NEBRASKA [US]; OHIO STATE INNOVATION FOUNDATION [US]) 23 June 2016 (2016-06-23)
paragraphs [0078]-[0079], [0103], [0112]-[0118], [0151]-[0158],;claims; figure 1; examples; sequences 11, 22
WO 2018094251 A1 (KASPAR BRIAN K [US]; FOUST KEVIN [US]) 24 May 2018 (2018-05-24)
paragraphs [0036]-[0037];;claims; figures; examples; sequence 2
GARG S K ET AL, "Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome", THE JOURNAL OF NEUROSCIENCE,Vol. 33, No. 34, 21 August 2013 (2013-08-21), page 13612-13620,
abstract, materials and methods, results;
WO 2014022582 A1 (NATIONWIDE CHILDRENS HOSPITAL [US]) 06 February 2014 (2014-02-06)
paragraphs [0019]-[0021], [0050]-[0051];;claims; examples
SONDHI D ET AL, "Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene", HUMAN GENE THERAPY,Vol. 25, No. 3, 01 March 2014 (2014-03-01), page 223-239,
abstract; materials and methods; results; discussion;
Special categories of cited documents:
document defining the general state of the art which is not considered to be of particular relevance
document cited by the applicant in the international application
earlier application or patent but published on or after the international filing date
document which may throw doubts on priority claim(s) or which is cited to establish the publication date of another citation or other special reason (as specified)
document referring to an oral disclosure, use, exhibition or other means
document published prior to the international filing date but later than the priority date claimed
later document published after the international filing date or priority date and not in conflict with the application but cited to understand the principle or theory underlying the invention
document of particular relevance; the claimed invention cannot be considered novel or cannot be considered to involve an inventive step when the document is taken alone
document of particular relevance; the claimed invention cannot be considered to involve an inventive step when the document is combined with one or more other such documents, such combination being obvious to a person skilled in the art
document member of the same patent family


Patent document cited in search report Publication date
Patent family member(s) Publication date
WO 2016100575 A1
23 June 2016
AU 2015364636 A1
CA 2967468 A1
CN 107249646 A
EP 3233131 A1
JP 2018500311 A
US 2019032078 A1
WO 2016100575 A1
01 June 2017
23 June 2016
13 October 2017
25 October 2017
11 January 2018
31 January 2019
23 June 2016
WO 2018094251 A1
24 May 2018
AU 2017362491 A1
BR 112019009834 A2
CA 3044291 A1
CL 2019001354 A1
CN 110325219 A
EP 3541429 A1
JP 2020503265 A
KR 20190126047 A
WO 2018094251 A1
06 June 2019
08 October 2019
24 May 2018
13 September 2019
11 October 2019
25 September 2019
30 January 2020
08 November 2019
24 May 2018
WO 2014022582 A1
06 February 2014
AU 2013296425 A1
AU 2018226501 A1
BR 112015002168 A2
CA 2880653 A1
DK 2879719 T3
EP 2879719 A1
EP 3415167 A1
ES 2684222 T3
JP 6314138 B2
JP 2015525783 A
JP 2018115196 A
JP 2019116493 A
PT 2879719 T
US 2015252384 A1
US 2017216458 A1
US 2019099503 A1
US 2019134225 A1
US 2019134226 A1
US 2019151477 A1
US 2019262474 A1
US 2019269798 A1
US 2019336618 A1
WO 2014022582 A1
19 February 2015
27 September 2018
07 November 2017
06 February 2014
03 September 2018
10 June 2015
19 December 2018
01 October 2018
18 April 2018
07 September 2015
26 July 2018
18 July 2019
08 October 2018
10 September 2015
03 August 2017
04 April 2019
09 May 2019
09 May 2019
23 May 2019
29 August 2019
05 September 2019
07 November 2019
06 February 2014
Name and mailing address of the ISA/:
European Patent Office
P.B. 5818, Patentlaan 2, 2280 HV Rijswijk,
Telephone No. (+31-70)340-2040
Facsimile No. (+31-70)340-3016
Date of the actual completion of the international search:
14 May 2020
Date of mailing of the international search report:
27 May 2020
Authorized officer:
Sommer, Birgit
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