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Targeted corrective gene conversion (TCGC): Molecular systems for improved correction of gene mutations in muscle

Journal Article


Abstract

  • Targeted corrective gene conversion (TCGC) holds much

    promise as a future therapy for many hereditary diseases in

    humans, but there still remain significant impediments to

    effective mutation correction and it is clear that significant

    work remains to improve TCGC to levels where it can be considered for translation to the clinical setting. Nevertheless,

    mutation correction frequencies varying between 0.0001%

    and 40% have been reported using chimeraplasty, oligoplasty, triplex-forming oligonucleotides, and small corrective

    PCR amplicons. W

Authors

  •   Kapsa, Robert M. I.
  •   Razal, Joselito M. (external author)
  •   Quigley, Anita F. (external author)
  •   Kita, Magdalena
  •   Todaro, Marian (external author)
  •   Bissonauth, Luveen (external author)
  •   Shepherd, Roderick L. (external author)
  •   Moulton, Simon E. (external author)
  •   Cook, Mark J. (external author)
  •   Spiccia, Leone (external author)
  •   Officer, David L.
  •   Clark, Graeme M. (external author)
  •   Wallace, Gordon G.

Publication Date

  • 2009

Citation

  • Kapsa, R., Razal, J., Quigley, A., Kita, M., Todaro, M., Bissonauth, L., Shepherd, R., Moulton, S., Cook, M. J., Spiccia, L., Officer, D. L., Clark, G. & Wallace, G. G. (2009). Targeted corrective gene conversion (TCGC): Molecular systems for improved correction of gene mutations in muscle. In 6th Australasian Gene Therapy Society Meeting, 29 Apr- 1 May, Sydney. Journal of Gene Medicine, 11 (9), 856-856.

Ro Metadata Url

  • http://ro.uow.edu.au/aiimpapers/740

Number Of Pages

  • 0

Start Page

  • 856

End Page

  • 856

Volume

  • 11

Issue

  • 9

Abstract

  • Targeted corrective gene conversion (TCGC) holds much

    promise as a future therapy for many hereditary diseases in

    humans, but there still remain significant impediments to

    effective mutation correction and it is clear that significant

    work remains to improve TCGC to levels where it can be considered for translation to the clinical setting. Nevertheless,

    mutation correction frequencies varying between 0.0001%

    and 40% have been reported using chimeraplasty, oligoplasty, triplex-forming oligonucleotides, and small corrective

    PCR amplicons. W

Authors

  •   Kapsa, Robert M. I.
  •   Razal, Joselito M. (external author)
  •   Quigley, Anita F. (external author)
  •   Kita, Magdalena
  •   Todaro, Marian (external author)
  •   Bissonauth, Luveen (external author)
  •   Shepherd, Roderick L. (external author)
  •   Moulton, Simon E. (external author)
  •   Cook, Mark J. (external author)
  •   Spiccia, Leone (external author)
  •   Officer, David L.
  •   Clark, Graeme M. (external author)
  •   Wallace, Gordon G.

Publication Date

  • 2009

Citation

  • Kapsa, R., Razal, J., Quigley, A., Kita, M., Todaro, M., Bissonauth, L., Shepherd, R., Moulton, S., Cook, M. J., Spiccia, L., Officer, D. L., Clark, G. & Wallace, G. G. (2009). Targeted corrective gene conversion (TCGC): Molecular systems for improved correction of gene mutations in muscle. In 6th Australasian Gene Therapy Society Meeting, 29 Apr- 1 May, Sydney. Journal of Gene Medicine, 11 (9), 856-856.

Ro Metadata Url

  • http://ro.uow.edu.au/aiimpapers/740

Number Of Pages

  • 0

Start Page

  • 856

End Page

  • 856

Volume

  • 11

Issue

  • 9