Title:
NUCLEIC ACID MOLECULE TARGETING MITF GENE AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2021/249064
Kind Code:
A1
Abstract:
Provided is a small activating nucleic acid molecule, which is a nucleic acid molecule targeting the MITF gene and at least contains a first oligonucleotide chain. Also provided are a composition or preparation containing the small activating nucleic acid molecule and the use thereof. The use of the small activating nucleic acid molecule or the composition or preparation containing the small activating nucleic acid molecule can activate or up-regulate the expression of the MITF gene in cells, and treat diseases or conditions related to the insufficient or reduced expression of the MITF protein.
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Inventors:
LI LONGCHENG (CN)
KANG MOORIM (CN)
KANG MOORIM (CN)
Application Number:
PCT/CN2021/091834
Publication Date:
December 16, 2021
Filing Date:
May 06, 2021
Export Citation:
Assignee:
RACTIGEN THERAPEUTICS (CN)
International Classes:
C12N15/113; A61K31/713; A61P35/00
Foreign References:
| CN109071603A | 2018-12-21 | |||
| CN102429894A | 2012-05-02 | |||
| CN110959042A | 2020-04-03 | |||
| CN106032532A | 2016-10-19 |
Other References:
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DELL'ANNA, M. L.M. PICARDO: "A review and a new hypothesis for non-immunological pathogenetic mechanisms in vitiligo", PIGMENT CELL RES, vol. 19, 2006, pages 406 - 11
SEHGAL, V N.G. SRIVASTAVA: "Vitiligo: compendium of clinico-epidemiological features", INDIAN J DERMATOL VENEREOL LEPROL, vol. 73, 2007, pages 149 - 56
KOTOBUKI, YA. TANEMURAL. YANGS. ITOIM. WATAYA-KANEDAH. MUROTAM. FUJIMOTOS. SERADAT. NAKAI. KATAYAMA: "Dysregulation of melanocyte function by Th17-related cytokines: significance of Thl7 cell infiltration in autoimmune vitiligo vulgaris", PIGMENT CELL MELANOMA RES, vol. 25, 2012, pages 219 - 30
KEMP, E. H.N. G. GAVALASD. J. GAWKRODGERA. P. WEETMAN: "Autoantibody responses to melanocytes in the depigmenting skin disease vitiligo", AUTOIMMUN REV, vol. 6, 2007, pages 138 - 42, XP005876573, DOI: 10.1016/j.autrev.2006.09.010
DELL'ANNA, M. L.M. OTTAVIANIV. ALBANESIA. P. VIDOLING. LEONEC. FERRAROA. COSSARIZZAL. ROSSIM. PICARDO: "Membrane lipid alterations as a possible basis for melanocyte degeneration in vitiligo", J INVEST DERMATOL, vol. 127, 2007, pages 1226 - 33
KUMAR, R.D. PARSADA. J. KANWAR: "Role of apoptosis and melanocytorrhagy: a comparative study of melanocyte adhesion in stable and unstable vitiligo", BR J DERMATOL, vol. 164, 2011, pages 187 - 91
HEMESATH, T. J.E. STEINGRIMSSONG. MCGILLM. J. HANSENJ. VAUGHTC. A. HODGKINSONH. ARNHEITERN. G. COPELANDN. A. JENKINSD. E. FISHER: "microphthalmia, a critical factor in melanocyte development, defines a discrete transcription factor family", GENES DEV, vol. 8, 1994, pages 2770 - 80, XP000909088
LEVY, C.M. KHALEDD. E. FISHER: "MITF: master regulator of melanocyte development and melanoma oncogene", TRENDS MOL MED, vol. 12, 2006, pages 406 - 14, XP028058641, DOI: 10.1016/j.molmed.2006.07.008
KWINTER, J.J. PELLETIERA. KHAMBALIAE. POPE: "High-potency steroid use in children with vitiligo: a retrospective study", JAM ACAD DERMATOL, vol. 56, 2007, pages 236 - 41, XP005860328, DOI: 10.1016/j.jaad.2006.08.017
IANNELLA, G.A. GRECOD. DIDONAB. DIDONAG. GRANATAA. MANNOB. PASQUARIELLOG. MAGLIULO: "Vitiligo: Pathogenesis, clinical variants and treatment approaches", AUTOIMMUN REV, vol. 15, 2016, pages 335 - 43, XP029432853, DOI: 10.1016/j.autrev.2015.12.006
LIM, H. W., P. E. GRIMES, O. AGBAI, I. HAMZAVI, M. HENDERSON, M. HADDICAN, R. V. LINKNER, M. LEBWOHL: "Afamelanotide and narrowband UV-B phototherapy for the treatment of vitiligo: a randomized multicenter trial", JAMA DERMATOL, vol. 151, 2015, pages 42 - 50, XP009186730, DOI: 10.1001/jamadermatol.2014.1875
GRIMES, P. E.I. HAMZAVIM. LEBWOHLJ. P. ORTONNEH. W. LIM: "The efficacy of afamelanotide and narrowband UV-B phototherapy for repigmentation of vitiligo", JAMA DERMATOL, vol. 149, 2013, pages 68 - 73, XP008162255, DOI: 10.1001/2013.jamadermatol.386
PICARDO, M., M. L. DELL'ANNA, K. EZZEDINE, I. HAMZAVI, J. E. HARRIS, D. PARSAD, A. TAIEB.: "Vitiligo", NAT REV DIS PRIMERS, vol. 1, 2015, pages 15011
GAUTHIER, YM. CARIO ANDREA. TAIEB: "A critical appraisal of vitiligo etiologic theories. Is melanocyte loss a melanocytorrhagy?", PIGMENT CELL RES, vol. 16, 2003, pages 322 - 32
DELL'ANNA, M. L.M. PICARDO: "A review and a new hypothesis for non-immunological pathogenetic mechanisms in vitiligo", PIGMENT CELL RES, vol. 19, 2006, pages 406 - 11
SEHGAL, V N.G. SRIVASTAVA: "Vitiligo: compendium of clinico-epidemiological features", INDIAN J DERMATOL VENEREOL LEPROL, vol. 73, 2007, pages 149 - 56
KOTOBUKI, YA. TANEMURAL. YANGS. ITOIM. WATAYA-KANEDAH. MUROTAM. FUJIMOTOS. SERADAT. NAKAI. KATAYAMA: "Dysregulation of melanocyte function by Th17-related cytokines: significance of Thl7 cell infiltration in autoimmune vitiligo vulgaris", PIGMENT CELL MELANOMA RES, vol. 25, 2012, pages 219 - 30
KEMP, E. H.N. G. GAVALASD. J. GAWKRODGERA. P. WEETMAN: "Autoantibody responses to melanocytes in the depigmenting skin disease vitiligo", AUTOIMMUN REV, vol. 6, 2007, pages 138 - 42, XP005876573, DOI: 10.1016/j.autrev.2006.09.010
DELL'ANNA, M. L.M. OTTAVIANIV. ALBANESIA. P. VIDOLING. LEONEC. FERRAROA. COSSARIZZAL. ROSSIM. PICARDO: "Membrane lipid alterations as a possible basis for melanocyte degeneration in vitiligo", J INVEST DERMATOL, vol. 127, 2007, pages 1226 - 33
KUMAR, R.D. PARSADA. J. KANWAR: "Role of apoptosis and melanocytorrhagy: a comparative study of melanocyte adhesion in stable and unstable vitiligo", BR J DERMATOL, vol. 164, 2011, pages 187 - 91
HEMESATH, T. J.E. STEINGRIMSSONG. MCGILLM. J. HANSENJ. VAUGHTC. A. HODGKINSONH. ARNHEITERN. G. COPELANDN. A. JENKINSD. E. FISHER: "microphthalmia, a critical factor in melanocyte development, defines a discrete transcription factor family", GENES DEV, vol. 8, 1994, pages 2770 - 80, XP000909088
LEVY, C.M. KHALEDD. E. FISHER: "MITF: master regulator of melanocyte development and melanoma oncogene", TRENDS MOL MED, vol. 12, 2006, pages 406 - 14, XP028058641, DOI: 10.1016/j.molmed.2006.07.008
KWINTER, J.J. PELLETIERA. KHAMBALIAE. POPE: "High-potency steroid use in children with vitiligo: a retrospective study", JAM ACAD DERMATOL, vol. 56, 2007, pages 236 - 41, XP005860328, DOI: 10.1016/j.jaad.2006.08.017
IANNELLA, G.A. GRECOD. DIDONAB. DIDONAG. GRANATAA. MANNOB. PASQUARIELLOG. MAGLIULO: "Vitiligo: Pathogenesis, clinical variants and treatment approaches", AUTOIMMUN REV, vol. 15, 2016, pages 335 - 43, XP029432853, DOI: 10.1016/j.autrev.2015.12.006
LIM, H. W., P. E. GRIMES, O. AGBAI, I. HAMZAVI, M. HENDERSON, M. HADDICAN, R. V. LINKNER, M. LEBWOHL: "Afamelanotide and narrowband UV-B phototherapy for the treatment of vitiligo: a randomized multicenter trial", JAMA DERMATOL, vol. 151, 2015, pages 42 - 50, XP009186730, DOI: 10.1001/jamadermatol.2014.1875
GRIMES, P. E.I. HAMZAVIM. LEBWOHLJ. P. ORTONNEH. W. LIM: "The efficacy of afamelanotide and narrowband UV-B phototherapy for repigmentation of vitiligo", JAMA DERMATOL, vol. 149, 2013, pages 68 - 73, XP008162255, DOI: 10.1001/2013.jamadermatol.386
Attorney, Agent or Firm:
TAHOTA LAW FIRM (CN)
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