Types of disorder gene thereapy can and
cannot treat
Can
treat:
-
all
forms of diseases can be treated to a various degree by altering gene expression
-
heal hereditary
diseases such as
o
muscular
distrophy
o
cystic
fibrosis
o
hemophilia
o
diabetes
type I
o
metabolic
diseases (phenylketonuria etc.)
o
lysosomal
storage disorders (mucopolysaccharosis, Gaucher etc..)
-
diseases
that are genetically predisposed, but also depend on external factors
such as
o
cancer
o
cardiovascular
failures
o
neurodegenerative
disorders (ALS, MS, Alzheimer's, Parkinson's etc)
-
purely
accidental (acquired) disorders such as
o
traumatic
injuries (bone fractures, wounds, burns)
o
ischemias
(tissue necrosis due to interrupoted blood supply)
o
infections
How gene therapy can be used to treat
ADA-deficient SCID
-
Adenosine
deaminase (ADA) deficiency is inherited as an autosomal recessive disorder. Defects in the ADA
gene can leads to absent or diminished ADA enzyme activity in all tissues of
the body.
-
The
reversing of this genetic defect by replacing the defective gene may be
possible by means of gene therapy.
-
The
newly isolated gene
may then be used for treatment by inserting it into the cells whose function is dependent on that gene.
The gene transfer
method used must be highly efficient and able to deliver the corrective gene to
the proper cells to restore its normal function.
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