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Ti li u chia s t i: wWw.SinhHoc.edu.vn Theo thong ke ngi co khoang 4000 benh do di truyen. Trong o co mot lng ln benh di truyen la do sai hong cua mot gen, tham ch cua mot base duy nhat. Phan ln cac benh nay eu la benh hiem ngheo, kho ieu tr. Tuy nhien s phat trien cua ky thuat di truyen m ra hy vong cho nhng benh nhan b benh di truyen
* Ngay 14 9 1990, nhom nghien cu tai hoc vien sc khoe quoc gia my a tien hanh chuoi th nghiem lieu phap gene au tien cho be Ashanti Desilva 4 tuoi. Sinh ra vi can benh di truyen hiem ngheo co ten la SCID (thieu nang mien dch hon hp cap ). Co be thieu he thong mien dch va co the b ton thng *khi mac phai bat ky mam benh phc tap, mot Lieu phap gene ngi la rat nao. so cong nghe can c phat trien va nhng can benh can c hieu biet hn trc khi lieu phap gene co the c s dung mot cach thch * Tuy nhien, khong the phu nhan rang, ngay nay ang. lieu phap gene la mot lnh vc khong lo vi c hoi sinh li ln cho cac nha au t co hieu biet. Bang chng la s phat trien cua cac nghien cu ve lieu phap gene trong hn mot thap ky qua.
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II. NH NGHA.
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Lieu phap gene la phng phap dung ky thuat di truyen e a gene lanh vao c the thay the cho gene benh hay a gene can thiet nao o thay vao v tr gene b sai hong e at c muc tieu cua lieu
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Bien phap th nhat (a gene lanh vao c the thay the gene benh) la cach giai quyet triet e, v gene lanh c a vao ung v tr cua no tren bo gene va chu tac ong bnh thng
nao o thay vao v tr gene b sai hong) th de thc hien hn nhng khi a them mot gen can thiet vao bo gene, ngi ta khong lam chu c v tr gan xen cua oan mi c a vao, ieu nay dan ti mot ket qua khong Gene mi a vao khong nam lng phc hp ieu hoa=> bieu trong trc c: hien tuy tien, khong ung ni, ung luc hoac khong c bieu hien.a vao gay nhng Gene mi bieu hien khong mong muon Gene gan vao mot v tr co kha nang hoat hoa mot gene tien ung th dan en s bieu
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* au tien, phai co ban sao ay u cua gene thch hp vi nhng trnh t ieu hoa thch hp ( trnh t khi ong). * Th hai, phai la chon ky thuat ch. * Th ba, phng phap ng dung lieu phap gen. Co the ng dung trc tiep vi benh nhan hoac tren cac te bao a tach ra khoi ngi benh.
V. TIEU CHUAN S DUNG LIEU PHAP GENE CHO BENH DI TRUYEN LY TNG.
1. Nguyen nhan gay benh phai do sai hong cua mot gene duy nhat. hng en 2. Benh ch anh
mot loai te bao duy nhat va te bao phai de tach. 3. Gen gay benh phai c xac nh va c tao dong. 4. S bieu hien ieu hoa cua nhng gen nay khong oi hoi sa cha nhng sai hong.
VI.Ti li LOAI GENtS DUNG TRONG LIEU CAC u chia s i: wWw.SinhHoc.edu.vn PHAP GEN Gen tr lieu la nhng oan oliosaccari t
Ti li u chia s t i: wWw.SinhHoc.edu.vn Cac gene hoat ong bnh thng (gene lanh): co the a vao te bao e thay the gene hong, gene mat chc nang, khoi phuc hoat ong bnh thng cua te bao va s song cua c the. Cac gene co kha nang ma hoa mot protein ac hieu: khi a vao te bao song co kha nang tao ra cac loai protein ac hieu. Cac protein ac hieu co the c che hoat ong cua mot gene khac trong te bao, km ham kha nang phan chia cua te bao hoac Nhng gene khi a vao te bao hoat ong ong gay chet te bao b benh. thi vi cac gene benh lam han che tac ong cua gene benh hoac ho tr, bu ap cho cac gene b hong bat hoat: c a vao te bao e thay the Gene cho mot gene lanh nao o nham han che san pham khong can thiet cua gene lanh hoac tao ra cho te bao mot lieu cung mi, co tac dung oan lai Gene tr trang thai co the la nhng chong benh tat. oligonucleotide co tac dung km ham hoat ong
cua gene b hong, b ot bien trong te bao.
VII. CAC PHNG PHAP S DUNG TRONG LIEU PHAP GENE: e cha benh bang lieu phap gene, ngi ta thng s dung 1 trong 3 cach sau: 1. In vivo 2. Ex vivo 3. In situ
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Ti li u chia s t i: wWw.SinhHoc.edu.vn
en nay co 533 th nghiem lieu phap gene dien ra tren the gii. Vi 407 th nghiem dien ra my, co 322 th nghiem trong ieu tr benh ung th, chiem 62,28%, 37 th nghiem trong ieu tr HIV chiem 6,94%, ngoai ra cac th nghiem vi cac benh
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3. Benh ung th.
* Ung th la s phat trien khong bnh thng cua te bao, do sai lech hoac ot bien mot hoac mot so gene trong te bao. Cac te bao ung th co qua trnh phan chia vo to chc, khong chu s kiem soat cua s chet theo chng trnh (apoptosis), tao nentr mi nhat hien * Ky thuat ieu cac dang khoi u.
nay la dung lieu phap gene in vivo va ex vivo, bang ky thuat vi tiem ngi ta a thang vao gia khoi u cac vector mang gene tr lieu tng ng e c che hoac tieu diet te bao ung th.
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Ti li u chia s t i: wWw.SinhHoc.edu.vn
4. Benh thieu mau hong cau li liem. Benh thieu mau hong cau li liem SCA (sickle cell anemia) la benh do ot bien gene globin lam thay oi chc nang cua protein, xuat hien hemoglobin S iem ot bien va cac dang ot bien a c nghien cu ky, tuy nhien s dung lieu phap gen cha SCA ngi thu c ket qua rat han che.
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S dung cac vect lieu phap e chuyen cac gene tr lieu vao te bao nham han che kha nang xam nhiem va tai ban cua HIV Kch hoat he thong gene cua te bao, tang cng mien dch chong s phat trien cua HIV/AIDS S dung cac gene t sat ced a bien oi, lam cho te bao b chet khi co HIV xam nhiem. Tao cac DNA vaccine phong chong nhiem HIV
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Gii i: wWw.SinhHoc.edu.vn Ti li u chia s tthieu chung ve lieu phap gen nh ngha Cac loai lieu phap gen C s cua lieu phap gen
Cac loai gen s dung trong lieu pha Cac phng phap s dung trong lieu phap gen
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