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Title:

GENE THERAPY ''

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See Undercover genes slip into the brain at NewScientist.com (March 20, 2003) ... designed to match the RNA copied from a ... Human Genome Project Information ... – PowerPoint PPT presentation

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Title: GENE THERAPY ''


1
????????(GENE THERAPY)?.??.???????
??????????????
2
???????? (GENE THERAPY)
  • ??????????????????????????????????????????????????
    ???????????????
  • ??????????
  • ?????????????????????????????????????
    ??????????????
  • ?????????????????????????????????? homologous
    recombination
  • ??????? Point mutation ?????????????????
  • ??????????????????????

3
Gene Therapy Clinical Trials Worldwide1989-2004
An Overview
  • ???? 1989, Rosenberg ?????????????????????????????
    ????? ?????????????????????????? NeoR
    ???????????????????????????
  • ????????? Clinical Trials ??????????????????????
    31 ?????? 2004 ??? 918 ???????? ?? 24 ?????? (2/3
    ?? USA)
  • Cancers gt Inherited monogenic dieseases gt
    Cardiovascular diseases
  • Viral vectors (retrovirus and adenovirus)
    ???????????????????????????????? (75)

4
(No Transcript)
5
????????????????????????????????
6
(No Transcript)
7
???????????????????????(gt100 ???)
8
Vectors Used in Clinical Trials
9
??????????????????????????
  • ??????????????????
  • 1999 ??????????? 18 ?? ???????????????????????????
    ?????? Adenovirus ?????????? vector
  • 2000 ????????????????????????????????? SCID-X1
  • 2002 ???? 2/10 ????????????? SCID-X1 ???????????
    ??????????????????????
  • 2003 ?????????????????? Retrovirus ????????

10
?????????????????????
  • ?????????????????????????? ???????????????????????
    ???????????????????
  • ??????????????????????????????????????
  • ??????????? ??????????????? ??????????????????????
    ???????????????? ?????????????????????????????????
    ?
  • ??????????????????????????????????????????????????
    ??

11
Recent Developments in Gene Therapy Research
  • University of California, Los Angeles, research
    team gets genes into the brain using liposomes
    coated in a polymer call polyethylene glycol
    (PEG). The transfer of genes into the brain is a
    significant achievement because viral vectors are
    too big to get across the "blood-brain barrier."
    This method has potential for treating
    Parkinson's disease. See Undercover genes slip
    into the brain at NewScientist.com (March 20,
    2003).

12
  • RNA interference or gene silencing may be a new
    way to treat Huntington's. Short pieces of
    double-stranded RNA (short, interfering RNAs or
    siRNAs) are used by cells to degrade RNA of a
    particular sequence. If a siRNA is designed to
    match the RNA copied from a faulty gene, then the
    abnormal protein product of that gene will not be
    produced. See Gene therapy may switch off
    Huntington's at NewScientist.com (March 13, 2003).

13
  • New gene therapy approach repairs errors in
    messenger RNA derived from defective genes.
    Technique has potential to treat the blood
    disorder thalassaemia, cystic fibrosis, and some
    cancers. See Subtle gene therapy tackles blood
    disorder at NewScientist.com (October 11, 2002).

14
  • Researchers at Case Western Reserve University
    and Copernicus Therapeutics are able to create
    tiny liposomes 25 nanometers across that can
    carry therapeutic DNA through pores in the
    nuclear membrane. See DNA nanoballs boost gene
    therapy at NewScientist.com (May 12, 2002).
  • Sickle cell is successfully treated in mice. See
    Murine Gene Therapy Corrects Symptoms of Sickle
    Cell Disease from March 18, 2002, issue of The
    Scientist.

15
References
  • Gene therapy clinical trials worldwide 1989-2004
    an overview. J Gene Med 2004 6 597-602.
  • Gene Therapy. Human Genome Project Information
  • http//www.ornl.gov/sci/techresources/Human_Genome
    /medicine/genetherapy.shtml
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