Patent SG-11201809777V-A (SG)
Title: Mitochondrial delivery of recombinant nucleic acids
Inventor: PATZEL VOLKER
IPC: C12N15/113
Filed: 2017-05-08
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 11111111111111111111111111111111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\"\" WO 2017/192102 Al 09 November 2017 (09.11.2017) WIP0 I PCT (51) International Patent Classification: C12N 15/11 (2006.01) A61K 48/00 (2006.01) Declarations under Rule 4.17: C12N 15/113 (2010.01) — of inventorship (Rule 4.17(iv)) (21) International Application Number: Published: PCT/SG2017/050238 — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a)) (22) International Filing Date: 08 May 2017 (08.05.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 10201603628Q 06 May 2016 (06.05.2016) SG (71) Applicant: NATIONAL UNIVERSITY OF SINGA- PORE [SG/SG]; 21 Lower Kent Ridge Road, Singapore 119077 (SG). (72) Inventors: PATZEL, Volker; c/o National University of Singapore, Yong Lo Lin School of Medicine, Department _ of Microbiology & Immunology, 21 Lower Kent Ridge Road, Singapore 119077 (SG). CHATTERJEE, Kaustav; — c/o National University of Singapore, Yong Lo Lin School of Medicine, Department of Microbiology & Immunology, 21 Lower Kent Ridge Road, Singapore 119077 (SG). = = _ — (74) Agent: SPRUSON & FERGUSON (ASIA) PTE LTD; = = P.O. Box 1531, Robinson Road Post Office, Singapore 903031 (SG). = Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, (81) = = HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, = = = PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. = = = Designated States (unless otherwise indicated, for every (84) = kind of regional protection available): ARIPO (BW, GH, — GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, = UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, — 1-1 MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, N KM, ML, MR, NE, SN, TD, TG). © ,-1 ei (54) Title: MITOCHONDRIAL DELIVERY OF RECOMBINANT NUCLEIC ACIDS 1-1 ---- (57) : The present disclosure describes a nucleic acid delivery construct comprising at least one sense or antisense RNA IN subdomain of the human cytomegalovirus [32.7 RNA, wherein each subdomain is capable of localization within the mitochondria, for C::::) transport into mitochondria. Disclosed herein are also methods of enhancing mitochondrial gene function, or suppressing defective N mitochondrial gene function, or both, as well as methods of treating a mitochondrial disorder. C