Biomedical Engineering Reference
In-Depth Information
No.
Diseases
Defect
Incidence
Target
References
1.
Gene therapy for hereditary diseases
1.1
Severe combined
immunodeficiency
Adenosine deaminase
Rare
Bone marrow or T cells
[2]
1.2
Hemophilia
Clotting factor VIII and IX
1/10,000
Liver and muscle
[3]
1.3
Familial
hypercholesterolemia
Low-density lipoprotein receptor
1/1,000,000
Liver
[4]
1.4
Cystic fibrosis
Loss of cystic fibrosis transmembrane
conductance regulator (CFTR gene)
1/3000
Lung
[5]
1.5
Hemoglobinopathies
and globins
1/600
Red blood cell precursors
[6]
1.6
Gaucher's disease
Glucocerebrosidase
1/450
Liver
[7]
1.7
Inherited emphysema
1
Antitrypsin
1/3500
Lung, liver
[8]
1.8
Muscular dystrophy
Dystrophin
1/3500 males
Muscle
[9]
2.
Gene therapy for acquired genetic diseases
2.1
Cancer
Defects in tumor suppressors and/or
presence of oncogenic factors(p53 gene)
1 year
Liver, breast, lung,
pancreas, prostate, kidney
[10]
2.2
Parkinson's disease
Neurotransmitter Release, -amyloid
protein structural defect
1 million in
USA
Brain
[11]
2.3
Alzheimer's disease
Neurotransmitter release, -amyloid
protein structural defect
4 million in
USA
Brain
[12]
2.4
Cardiovascular gene
delivery
Blood vessels defect (VEGF DNA
inserted)
15 lacs in
USA
Blood vessels
[13]
2.5
Infectious diseases
Supressed immune response and liver
destruction
15 lacs in
USA
T cell, liver, macrophage,
antigen-presenting cells
[14]
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