Biologic treatments for hereditary diseases / 生物工程学报
Chinese Journal of Biotechnology
;
(12): 968-975, 2015.
Article
in Chinese
| WPRIM
| ID: wpr-240572
ABSTRACT
Hereditary disease, especially monogenic disease is one of the major causes for malformation and disability of children. Most hereditary diseases have no effective therapy besides clinical symptomatic treatment. Biological techniques targeting casual genes or related signaling genes, such as transgenic, RNA interfere, genome editing, have been successfully applied in treating some hereditary diseases. However, most biological, treatments were carried out in animals, further confirmation of the effectiveness and safety of these therapies, and development of more therapeutic approaches based on mechanisms are needed before clinical trials.
Full text:
Available
Index:
WPRIM (Western Pacific)
Main subject:
Therapeutics
/
Biological Therapy
/
Genetic Therapy
/
Disease Models, Animal
/
Genetic Diseases, Inborn
Limits:
Animals
/
Humans
Language:
Chinese
Journal:
Chinese Journal of Biotechnology
Year:
2015
Type:
Article
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