Title:
POLYAMIDE COMPOUND AND PHARMACEUTICAL COMPOSITION FOR TREATING MITOCHONDRIAL GENETIC DISEASES
Document Type and Number:
WIPO Patent Application WO/2012/133896
Kind Code:
A1
Abstract:
The purpose of the present invention is to provide a fundamental treatment method for mitochondrial genetic diseases caused by a mutation of mitochondrial DNA, and a pharmaceutical composition therefor. This problem can be solved by a polyamide compound which binds to a target double-stranded mitochondrial DNA which has a sense strand DNA consisting of the base sequence represented by 5'-ATGGCAGAGCCCGGTAATCGCATAA-3' and an antisense strand DNA consisting of the base sequence represented by 5'-TTATGCGATTACCGGGCTCTGCCAT-3'. This double-stranded mitochondrial DNA includes an A/T pair of A in position 1 of the sense strand and the complementary T, an A/T pair of A in position 8 of the sense strand DNA and the complementary T, a G/C pair of G in position 9 of the sense strand DNA and the complementary C, a G/C pair of G in position 14 of the sense strand DNA and the complementary C, or a T/A pair of T in position 15 of the sense strand DNA and the complementary A.
Inventors:
YANO TAKAMITSU (JP)
Application Number:
PCT/JP2012/058957
Publication Date:
October 04, 2012
Filing Date:
April 02, 2012
Export Citation:
Assignee:
YANO TAKAMITSU (JP)
International Classes:
C12N15/09; A61K31/785; A61P3/10; A61P3/12; A61P9/10; A61P13/12; A61P21/00; A61P25/00; A61P25/08; A61P27/02; A61P27/16; A61P43/00
Foreign References:
| JP2004538326A | 2004-12-24 | |||
| JP2011503005A | 2011-01-27 |
Other References:
TAKAMITSU YANO: "MELAS A3243G Hen'i-gata Mitochondria DNA to Mitochondria Tensha Shuketsu Inshi mTERF no Kino Mechanism", 2010 NENDO GAKUI RONBUN (HAKUSHI), 2010, pages 7 - 10, 57 - 101, XP008171137
SHAAG, A. ET AL.: "Mitochondrial encephalomyopathy associated with a novel mutation in the mitochondrial tRNAleu(uuR) gene (A3243T).", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 233, no. 3, 1997, pages 637 - 639, XP002297267
SENECA, S. ET AL.: "A new mitochondrial point mutation in the transfer RNALeu gene in a patient with a clinical phenotype resembling Kearns-Sayre syndrome.", ARCHIVES OF NEUROLOGY, vol. 58, no. 7, 2001, pages 1113 - 1118, XP055128152
BOSLEY, T.M. ET AL.: "Sporadic bilateral optic neuropathy in children: the role of mitochondrial abnormalities.", INVESTIGATIVE OPHTHALMOLOGY AND VISUAL SCIENCE, vol. 49, no. 12, 2008, pages 5250 - 5256, XP055128157
GOTO, Y. ET AL.: "A novel point mutation in the mitochondrial tRNALeu(UUR) gene in a family with mitochondrial myopathy.", ANNALS OF NEUROLOGY, vol. 31, no. 6, 1992, pages 672 - 675, XP055128159
CHEN, Y. ET AL.: "Mitochondrial gene mutations in gestational diabetes mellitus.", DIABETES RESEARCH AND CLINICAL PRACTICE, vol. 48, no. 1, 2000, pages 29 - 35, XP055128167
NISHIGAKI, Y. ET AL.: "A novel mitochondrial tRNALeu(UUR) mutation in a patient with features of MERRF and Kearns-Sayre syndrome.", NEUROMUSCULAR DISORDERS, vol. 13, no. 4, 2003, pages 334 - 340, XP005480815
MARIOTTI, C. ET AL.: "Defective respiratory capacity and mitochondrial protein synthesis in transformant cybrids harboring the tRNALeu(UUR) mutation associated with maternally inherited myopathy and cardiomyopathy.", JOURNAL OF CLINICAL INVESTIGATION, vol. 93, no. 3, 1994, pages 1102 - 1107, XP055128172
HOUSHMAND, M. ET AL.: "Fatal mitochondrial myopathy, lactic acidosis, and complex I deficiency associated with a heteroplasmic A-G mutation at position 3251 in the mitochondrial tRNALeu(UUR) gene.", HUMAN GENETICS, vol. 97, no. 3, 1996, pages 269 - 273, XP008170844
NATURE GENETICS, BRITAIN, vol. 15, 1997, pages 212 - 215
ADVANCED DRUG DELIVERY REVIEWS, vol. 49, 2001, pages 121 - 125
SHAAG, A. ET AL.: "Mitochondrial encephalomyopathy associated with a novel mutation in the mitochondrial tRNAleu(uuR) gene (A3243T).", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 233, no. 3, 1997, pages 637 - 639, XP002297267
SENECA, S. ET AL.: "A new mitochondrial point mutation in the transfer RNALeu gene in a patient with a clinical phenotype resembling Kearns-Sayre syndrome.", ARCHIVES OF NEUROLOGY, vol. 58, no. 7, 2001, pages 1113 - 1118, XP055128152
BOSLEY, T.M. ET AL.: "Sporadic bilateral optic neuropathy in children: the role of mitochondrial abnormalities.", INVESTIGATIVE OPHTHALMOLOGY AND VISUAL SCIENCE, vol. 49, no. 12, 2008, pages 5250 - 5256, XP055128157
GOTO, Y. ET AL.: "A novel point mutation in the mitochondrial tRNALeu(UUR) gene in a family with mitochondrial myopathy.", ANNALS OF NEUROLOGY, vol. 31, no. 6, 1992, pages 672 - 675, XP055128159
CHEN, Y. ET AL.: "Mitochondrial gene mutations in gestational diabetes mellitus.", DIABETES RESEARCH AND CLINICAL PRACTICE, vol. 48, no. 1, 2000, pages 29 - 35, XP055128167
NISHIGAKI, Y. ET AL.: "A novel mitochondrial tRNALeu(UUR) mutation in a patient with features of MERRF and Kearns-Sayre syndrome.", NEUROMUSCULAR DISORDERS, vol. 13, no. 4, 2003, pages 334 - 340, XP005480815
MARIOTTI, C. ET AL.: "Defective respiratory capacity and mitochondrial protein synthesis in transformant cybrids harboring the tRNALeu(UUR) mutation associated with maternally inherited myopathy and cardiomyopathy.", JOURNAL OF CLINICAL INVESTIGATION, vol. 93, no. 3, 1994, pages 1102 - 1107, XP055128172
HOUSHMAND, M. ET AL.: "Fatal mitochondrial myopathy, lactic acidosis, and complex I deficiency associated with a heteroplasmic A-G mutation at position 3251 in the mitochondrial tRNALeu(UUR) gene.", HUMAN GENETICS, vol. 97, no. 3, 1996, pages 269 - 273, XP008170844
NATURE GENETICS, BRITAIN, vol. 15, 1997, pages 212 - 215
ADVANCED DRUG DELIVERY REVIEWS, vol. 49, 2001, pages 121 - 125
Attorney, Agent or Firm:
MORITA Kenichi et al. (JP)
Ken-ichi Morita (JP)
Ken-ichi Morita (JP)
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