Title:
RECOMBINANT VECTORS COMPRISING ARYLSULFATASE A AND THEIR USES IN STEM CELL THERAPY FOR THE TREATMENT OF METACHROMATIC LEUKODYSTROPHY
Document Type and Number:
WIPO Patent Application WO/2020/177618
Kind Code:
A1
Abstract:
Provided are recombinant lentiviral vectors comprising an expression cassette comprising a nucleic acid construct expressing an ARSA gene. The vectors are useful in gene therapy for the treatment of Metachromatic Leukodystrophy. Provided are methods of producing the vectors. Provided are multipotent stem cells comprising the vectors. Also provided are methods of culturing the stem cells to maintain their multipotency.
Inventors:
LIAN QIZHOU (CN)
Application Number:
PCT/CN2020/077088
Publication Date:
September 10, 2020
Filing Date:
February 28, 2020
Export Citation:
Assignee:
VERSITECH LTD (CN)
International Classes:
A61K48/00; C12N15/867; A61P3/00; A61P25/28; C12N5/10
Domestic Patent References:
| WO2018071898A1 | 2018-04-19 |
Foreign References:
| CN108707627A | 2018-10-26 |
Other References:
ALESSANDRA BIFFI; EUGENIO MONTINI; LAURA LORIOLI; MARTINA CESANI; FRANCESCA FUMAGALLI; TIZIANA PLATI; CRISTINA BALDOLI; SABATA MAR: "Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy", SCIENCE, vol. 341, no. 6148, 23 August 2013 (2013-08-23), pages 1 - 6, XP008163598, ISSN: 0036-8075, DOI: 10.1126/science.1233158
WANG LI-CHENG，YANG LI，GUO LI-HUA，ZHANG GUO-WEI: "Progress in clinical trials of lentiviral vector-mediated gene therapy", HEALTH RESEARCH, vol. 36, no. 6, 31 December 2016 (2016-12-31), pages 637 - 640, XP055731872, ISSN: 1674-6449, DOI: 10.3969/jissn.1674-6449.2016.06.012
WANG LI-CHENG，YANG LI，GUO LI-HUA，ZHANG GUO-WEI: "Progress in clinical trials of lentiviral vector-mediated gene therapy", HEALTH RESEARCH, vol. 36, no. 6, 31 December 2016 (2016-12-31), pages 637 - 640, XP055731872, ISSN: 1674-6449, DOI: 10.3969/jissn.1674-6449.2016.06.012
Attorney, Agent or Firm:
LIU, SHEN & ASSOCIATES (CN)
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