Details on Curatives

New Curatives on Mitochondrial Disease (MELAS)

 Over extended research period, we have developed a new way of treatment which utilizing ML1 polyamide.

 Effectiveness of the curative has been confirmed on cells collected from the skin of MELAS patients.

 ML1 polyamide enters mitochondria of patient’s cells and promotes the previously-suppressed replication of normal copies of mitochondrial DNA. With the gradual increase of the ratio of normal mitochondrial DNA in patient’s cells, the amount of normal mitochondrial DNA copies eventually overwhelms that of the mutated DNA, restoring the original function of mitochondria.


Patent records are downloadable through the links below.


Pyrrole-imidazole polyamide, a synthetic DNA-binding compound, is effective at increasing levels of wild-type mtDNA in both cybrid cells and MELAS patient-derived fibroblast cells with the MELAS A3243G mutation by a selective promotion of wild-type replication
Takamitsu Yano, Nono Tomita, Takuya Ueda, Richard H. Haas,
Vol 13 (6) 924-925, 2013, MITOCHONDRION
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MELAS A3243G mutant mitochondrial DNA and the functional mechanism of mitochondrial transcription termination factor, mTERF (Japanese)
Takamitsu Yano, The University of Tokyo, Doctoral Thesis, Dec. 2010
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Loss of mutant mitochondrial DNA harboring the MELAS A3243G mutation in human cybrid cells after cell-cell fusion with normal tissue-derived fibroblast cells
Takamitsu Yano, Masashi Tanaka, Noboru Fukuda, Takuya Ueda, Hiroki Nagase,
Vol 25 (1) 153-158, 2010, International Journal of Molecular Medicine
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Related Conferences

  1. The selective replication activation of wild-type mtDNA by a polyamide compound in cybrid cells and MELAS patient-derived fibroblast cells
    International Symposium on Mitochondria 2013:
    The 13th Conference of Japanese Society of Mitochondrial Research and Medicine (J-mit) Nov. 2013 (Poster)
    Takamitsu Yano, Nono Tomita, Takuya Ueda, Richard H. Haas
  2. The novel therapeutic strategy for mitochondrial DNA disease: A selective promotion of wild-type mitochondrial DNA replication by a synthetic DNA-binding compound
    The Molecular Biology Society of Japan Dec. 2011 (Oral)
    Takamitsu Yano, Nono Tomita, Takeshi Wada, Takuya Ueda, Yu-ichi Goto
  3. Successful preferential propagation of wild‐type mitochondrial DNA in human cells harboring the mt tRNALeu(UUR) A3243G mutation by a synthetic DNA‐binding compound
    United Mitochondrial Disease Foundation, Mitochondrial Medicine 2011: Chicago Jun. 2011 (Oral)
    Takamitsu Yano, Nono Tomita, Takuya Ueda
  4. Preferential loss of mitochondrial DNA harboring the pathogenic mutation tRNALeu(UUR) 3243 A->G in human somatic cell hybrids
    The 7th conference of Japanese Society of Mitochondrial Research and Medicine (J-mit) Dec. 2005 (Oral)
    Takamitsu Yano, Masashi Tanaka, Takuya Ueda

New Curatives on Muscular Dystrophy (DMD)

 Currently, treatment of DMD is performed by inducing the phenomenon of “exon skipping” through drugs. While this treatment may be able to sustain the life of the patient, the patient is not able to live the same life as normal peers.

 The curative we are developing does not cause exon skipping in dystrophin gene. Preserving the length as much as possible, more functional dystrophin can be produced to aim for restoration of normal function of muscles.

 By enabling tailor-made treatment for each patient, higher quality of life can be achieved, such that patients can be liberated from beds and wheelchairs and live a life similar to that of a normal person. This is our ultimate goal of the research project on these drugs.

Let us bring the light of hope to those who are suffering from intractable diseases

Developing a new drug is a costly operation. Your generous support will be vital in our research and development efforts.

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