分子生物学分子生物学 (37).pdf
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1、 Chapter 13 Gene Therapy 13.5 Application of Gene Therapy 1.Adenosine deaminase(ADA)and purine nucleoside phosphorylase(PNP)Deficiency 2.Globin aplastic anemia and hemoglobinopathy 3.Hemophilia and other plasma protein deficiencies 4.Phenylpyruvateuria and other congenital metabolic defects 5.Le-Na(
2、Lesch-Nyhan)syndrome 6.Familial hypercholesterolemia 7.Cystic fibrosis 13.5.1 Gene therapy for genetic diseases The first case-adenosine deaminase deficiency Physiology:adenosine deaminase is an intracellular enzyme that plays an important role in nucleic acid metabolism,which makes deoxyadenosine d
3、eamination in lymphocytes become deoxyinosine,and finally metabolizes to uric acid and then discharged from the body.Pathology:adenosine deaminase deficiency-deoxyadenosine can not be deaminated-intracellular accumulation of deoxyadenosine-lymphocyte death-lack of immune function.Treatment basis The
4、 adenosine deaminase gene is located in the long arm of the chromosome 20 The adenosine deaminase gene was cloned in 1983 Retrovirus vector can be used to send the gene to lymphocytes lacking adenosine deaminase gene The newly entered adenosine deaminase gene can be expressed in lymphocytes that did
5、 not have it ADA deficiency French Anderson(NIH)French Anderson(NIH)LTR ADA SV40 neo LTR September 14,1990September 14,1990 Clinical application of gene therapyClinical application of gene therapy Ashanti de Silva AshantiAshanti waswas thethe firstfirst patientpatient toto bebe treatedtreated withwi
6、th genegene therapytherapy.InjectionsInjections repeatedrepeated 7 7 timestimes inin firstfirst 1010.5 5 monthsmonths ADAADA:1 1%2525%Treatment steps Extract the patients bone marrow,isolate lymphocytes The patients lymphocytes were mixed with the retroviral vector carrying the adenosine deaminase g
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