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Gene Therapy

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Academic year: 2021

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(1)

Gene Therapy

(2)

- has emerged in the late 20th century

- a potential approach to the treatment of genetic disorders which are resistant to traditional therapies

- to cure genetic diseases by introducing new genetic instructions into the tissues of patients in order to compensate for abnormal or missing genes or to convey a new function

- cancer, cardiovascular, infectious, eye or lung diseases

- Many success stories in the recent years (ALD, inherited retinal diseases, SCID)

Gene Therapy

Example: Hemophilia A gene therapy

http://www.nwabr.org/studentbiotech/winners/studentwork/20 07/WB_BA_TRONGTHAM/1_index_2.htm

(3)

Gene Therapy - vectors

http://www.biochem.arizona.edu/classes/bioc471/p ages/Lecture25/Lecture25.html

- Viral vs non-viral vectors - Viral:

 Ad

 AAV

 Lentivirus

 Retrovirus

 SFV

- Non-viral vectors:

 Liposomes

 Polymer-based nanoparticles

 Dendrimers

(4)

- the most commonly used viral vector in gene therapy studies

- high transfection efficiency in a wide variety of cells

- can be amplified at very high titers.

fiber knob

shaf

penton RGD motif

hexon pXI

Adenovirus (Ad)

 Systemic administration

• Elicit strong immune responses, hepatotoxicity

• Show decreased blood circulation

Referanslar

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