Title:
TRANS-SPLICING RIBOZYME THAT IS SPECIFIC TO RHODOPSIN TRANSCRIPTOME, AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/019706
Kind Code:
A1
Abstract:
The present invention relates to a trans-splicing ribozyme targeting a rhodopsin transcriptome, and a use thereof for treating and/or preventing retinitis pigmentosa.
Inventors:
LEE SEONG-WOOK (KR)
KIM JI HYUN (KR)
HAN SEUNG RYUL (KR)
KIM JI HYUN (KR)
HAN SEUNG RYUL (KR)
Application Number:
PCT/KR2021/009555
Publication Date:
January 27, 2022
Filing Date:
July 23, 2021
Export Citation:
Assignee:
RZNOMICS INC (KR)
International Classes:
C12N15/113; A61K31/713; A61P27/02; C12N15/86
Foreign References:
| KR20100052070A | 2010-05-19 | |||
| KR20100024055A | 2010-03-05 |
Other References:
ADELINE BERGER, STéPHANIE LORAIN, CHARLèNE JOSéPHINE, MELISSA DESROSIERS, CéCILE PECCATE, THOMAS VOIT, LUIS GA: "Repair of Rhodopsin mRNA by Spliceosome-Mediated RNA Trans -Splicing: A New Approach for Autosomal Dominant Retinitis Pigmentosa", MOLECULAR THERAPY, vol. 23, no. 5, 1 May 2015 (2015-05-01), US , pages 918 - 930, XP055607775, ISSN: 1525-0016, DOI: 10.1038/mt.2015.11
KOHLER, U. AYRE, B.G. GOODMAN, H.M. HASELOFF, J.: "Trans-splicing ribozymes for targeted gene delivery", JOURNAL OF MOLECULAR BIOLOGY, vol. 285, no. 5, 5 February 1999 (1999-02-05), United Kingdom , pages 1935 - 1950, XP004464306, ISSN: 0022-2836, DOI: 10.1006/jmbi.1998.2447
CHAKRABORTY DIBYENDU, WHALEN PATRICK, LEWIN ALFRED S., NAASH MUNA I.: "In vitro Analysis of Ribozyme-mediated Knockdown of an ADRP Associated Rhodopsin Mutation", RETINAL DEGENERATIVE DISEASES: ADVANCES INEXPERIMENTAL MEDICINE AND BIOLOGY, vol. 613, 1 January 2008 (2008-01-01), US , pages 97 - 106, XP055888929, ISSN: 0065-2598, DOI: 10.1007/978-0-387-74904-4_10
PROG RETIN EYE RES, vol. 62, January 2018 (2018-01-01), pages 1 - 23
CHOI, V. W. ET AL.: "AAV hybrid serotypes: improved vectors for gene delivery", CURR GENE THER., vol. 5, no. 3, 2005, pages 299 - 310, XP002462124, DOI: 10.2174/1566523054064968
WU ET AL., MOLECULAR THERAPY, vol. 14, no. 3, pages 316 - 327
KOHLER, U. AYRE, B.G. GOODMAN, H.M. HASELOFF, J.: "Trans-splicing ribozymes for targeted gene delivery", JOURNAL OF MOLECULAR BIOLOGY, vol. 285, no. 5, 5 February 1999 (1999-02-05), United Kingdom , pages 1935 - 1950, XP004464306, ISSN: 0022-2836, DOI: 10.1006/jmbi.1998.2447
CHAKRABORTY DIBYENDU, WHALEN PATRICK, LEWIN ALFRED S., NAASH MUNA I.: "In vitro Analysis of Ribozyme-mediated Knockdown of an ADRP Associated Rhodopsin Mutation", RETINAL DEGENERATIVE DISEASES: ADVANCES INEXPERIMENTAL MEDICINE AND BIOLOGY, vol. 613, 1 January 2008 (2008-01-01), US , pages 97 - 106, XP055888929, ISSN: 0065-2598, DOI: 10.1007/978-0-387-74904-4_10
PROG RETIN EYE RES, vol. 62, January 2018 (2018-01-01), pages 1 - 23
CHOI, V. W. ET AL.: "AAV hybrid serotypes: improved vectors for gene delivery", CURR GENE THER., vol. 5, no. 3, 2005, pages 299 - 310, XP002462124, DOI: 10.2174/1566523054064968
WU ET AL., MOLECULAR THERAPY, vol. 14, no. 3, pages 316 - 327
Attorney, Agent or Firm:
MUHANN PATENT & LAW FIRM (KR)
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