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Gene therapy:
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Gene therapy is a technique which involves removal or manupulation or replacement of
defective gene with healthy one in order to treat genetic disorder
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The first gene therapy was
given in 1990 to a 4-year-old girl suffering from ADA deficiency
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Germline gene therapy:Germline therapy involves the modification of the genes inside germ or
gamete cells, which include sperm or ova
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That is the gene therapy done at site of infection of specific organ
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If adenosine is high it’ll by to its receptor ( adenosine receptor) that is predominantly found
in lymphocytes so increase in adenosine suppress the immune response
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The first trial for gene therapy was made in September 14, 1990 to a 4 year girl named
ashanki by William french andersonThese patients have non-functional lymphocytes and
they cannot mount immune responses against invading pathogens
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so anderson came up with a plan of gene therapy
Steps involved:
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The abnormal hematopoietic stem cells (containing the mutated gene) are extracted from
the blood or bone marrow of the of the child and sent to the laboratory where the they will
be fixed
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These copies are normal
genes and do not contain the mutation
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In the lab, doctors mix the virus, containing the normal gene, with the patient’s
hematopoietic stem cells
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The “corrected” hematopoietic stem cells are now able to make normal T cells
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Before the corrected hematopoietic stem cells are infused into the child, the child may or
may not receive conditioning, such as chemotherapy or other immuno-suppressant drugs
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The corrected hematopoietic stem cells are taken out of storage, and are given back to the
child in a simple IV infusion
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This gene is
responsible for encoding the CFTR protein, a chloride ion channel anchored in the plasma
membrane of lung cells, pancreatic cells, sweat and other exocrine glands
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Cystic fibrosis in male leads to infertility
It is a automatical recessive condition
The first trial for cystic fibrosis made in 1993
There were two type of method taken into consideration
Nebulized liposome mediated gene therapy and conjuncting a monoclonal antibody with
liposome to delivery to Target cell( Target drug delivery)
It is of invivo therapy
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Steps involved in invivo:
In vivo method of gene transfer involves the transfer of cloned genes directly into the
Tissues of the patient
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In case of viral vectors such type of cultured cells were often used which have been Infected
with the recombinant retrovirus in vitro to produce modified viral vectors Regularly
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The VPCs transfer the
gene to surrounding disease cells