{"id":7934,"date":"2024-08-20T08:40:26","date_gmt":"2024-08-20T01:40:26","guid":{"rendered":"https:\/\/jhasim1.com\/?p=7934"},"modified":"2024-08-10T21:38:24","modified_gmt":"2024-08-10T14:38:24","slug":"nghien-cuu-moi-tien-bo-trong-lieu-phap-chinh-sua-gen-crispr","status":"publish","type":"post","link":"https:\/\/jhasim1.com\/nghien-cuu-moi-tien-bo-trong-lieu-phap-chinh-sua-gen-crispr.html","title":{"rendered":"Ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p ch\u1ec9nh s\u1eeda gen CRISPR"},"content":{"rendered":"
Trong nh\u1eefng n\u0103m g\u1ea7n \u0111\u00e2y, ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p ch\u1ec9nh s\u1eeda gen CRISPR \u0111\u00e3 t\u1ea1o ra m\u1ed9t cu\u1ed9c c\u00e1ch m\u1ea1ng trong l\u0129nh v\u1ef1c y h\u1ecdc v\u00e0 c\u00f4ng ngh\u1ec7 sinh h\u1ecdc. CRISPR-Cas9, h\u1ec7 th\u1ed1ng ch\u1ec9nh s\u1eeda gen d\u1ef1a tr\u00ean CRISPR, \u0111\u00e3 tr\u1edf th\u00e0nh c\u00f4ng c\u1ee5 quan tr\u1ecdng cho c\u00e1c nghi\u00ean c\u1ee9u gen v\u00e0 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh. Nh\u1edd v\u00e0o ti\u1ebfn b\u1ed9 n\u00e0y, c\u00e1c nh\u00e0 khoa h\u1ecdc c\u00f3 kh\u1ea3 n\u0103ng thay \u0111\u1ed5i gen c\u1ee7a m\u1ed9t lo\u00e0i s\u1ed1ng m\u1ed9t c\u00e1ch ch\u00ednh x\u00e1c v\u00e0 hi\u1ec7u qu\u1ea3 h\u01a1n bao gi\u1edd h\u1ebft.<\/div>\n
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CRISPR-Cas9 l\u00e0 g\u00ec?<\/div>\n
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CRISPR-Cas9 l\u00e0 m\u1ed9t h\u1ec7 th\u1ed1ng ch\u1ec9nh s\u1eeda gen t\u1ef1 nhi\u00ean \u0111\u01b0\u1ee3c t\u00ecm th\u1ea5y trong vi khu\u1ea9n. CRISPR, vi\u1ebft t\u1eaft c\u1ee7a “Clustered Regularly Interspaced Short Palindromic Repeats” (l\u1eb7p l\u1ea1i ng\u1eafn xen k\u1ebd \u0111\u1ec1u \u0111\u1eb7n c\u00f3 t\u00ednh \u0111\u1ed1i x\u1ee9ng), l\u00e0 m\u1ed9t lo\u1ea1i DNA kh\u00f4ng m\u00e3 h\u00f3a \u0111\u01b0\u1ee3c t\u00ecm th\u1ea5y trong vi khu\u1ea9n v\u00e0 vi r\u00fat. C\u00f2n Cas9 l\u00e0 m\u1ed9t lo\u1ea1i enzyme c\u00f3 kh\u1ea3 n\u0103ng c\u1eaft DNA. Khi Cas9 \u0111\u01b0\u1ee3c k\u1ebft h\u1ee3p v\u1edbi m\u1ed9t \u0111o\u1ea1n RNA ch\u1ec9 d\u1eabn, n\u00f3 c\u00f3 th\u1ec3 nh\u1eadn di\u1ec7n v\u00e0 c\u1eaft ch\u00ednh x\u00e1c c\u00e1c \u0111o\u1ea1n DNA t\u01b0\u01a1ng \u1ee9ng trong t\u1ebf b\u00e0o s\u1ed1ng.<\/div>\n
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Cas9 \u0111\u01b0\u1ee3c k\u1ebft h\u1ee3p v\u1edbi m\u1ed9t \u0111o\u1ea1n RNA ch\u1ec9 d\u1eabn nh\u1eadn di\u1ec7n v\u00e0 c\u1eaft ch\u00ednh x\u00e1c c\u00e1c \u0111o\u1ea1n DNA t\u01b0\u01a1ng \u1ee9ng trong t\u1ebf b\u00e0o s\u1ed1ng.<\/figcaption><\/figure>\n<\/div>\n
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\u1ee8ng d\u1ee5ng c\u1ee7a CRISPR-Cas9<\/div>\n
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Ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p ch\u1ec9nh s\u1eeda gen CRISPR \u0111\u00e3 m\u1edf ra m\u1ed9t lo\u1ea1t \u1ee9ng d\u1ee5ng trong nghi\u00ean c\u1ee9u gen v\u00e0 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh. V\u1edbi kh\u1ea3 n\u0103ng ch\u1ec9nh s\u1eeda gen ch\u00ednh x\u00e1c v\u00e0 hi\u1ec7u qu\u1ea3, CRISPR-Cas9 c\u00f3 th\u1ec3 gi\u00fap ch\u00fang ta hi\u1ec3u r\u00f5 h\u01a1n v\u1ec1 vai tr\u00f2 c\u1ee7a c\u00e1c gen trong c\u01a1 th\u1ec3 s\u1ed1ng v\u00e0 t\u00ecm ra nh\u1eefng ph\u01b0\u01a1ng ph\u00e1p m\u1edbi \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb c\u00e1c b\u1ec7nh di truy\u1ec1n. D\u01b0\u1edbi \u0111\u00e2y l\u00e0 m\u1ed9t s\u1ed1 \u1ee9ng d\u1ee5ng quan tr\u1ecdng c\u1ee7a CRISPR-Cas9:<\/div>\n
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1. Nghi\u00ean c\u1ee9u gen<\/div>\n
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C\u00e1c nh\u00e0 nghi\u00ean c\u1ee9u t\u1ea1o ra c\u00e1c m\u00f4 h\u00ecnh b\u1ec7nh trong ph\u00f2ng th\u00ed nghi\u1ec7m nh\u1edd CRISPR-Cas9<\/figcaption><\/figure>\n<\/div>\n
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CRISPR-Cas9 cho ph\u00e9p c\u00e1c nh\u00e0 khoa h\u1ecdc ch\u1ec9nh s\u1eeda gen c\u1ee7a c\u00e1c sinh v\u1eadt s\u1ed1ng m\u1ed9t c\u00e1ch ch\u00ednh x\u00e1c v\u00e0 nhanh ch\u00f3ng. \u0110i\u1ec1u n\u00e0y gi\u00fap ch\u00fang ta hi\u1ec3u r\u00f5 h\u01a1n v\u1ec1 vai tr\u00f2 c\u1ee7a c\u00e1c gen trong qu\u00e1 tr\u00ecnh ph\u00e1t tri\u1ec3n v\u00e0 ch\u1ee9c n\u0103ng c\u1ee7a ch\u00fang trong c\u01a1 th\u1ec3 s\u1ed1ng. Nh\u1edd v\u00e0o CRISPR-Cas9, c\u00e1c nh\u00e0 nghi\u00ean c\u1ee9u c\u00f3 th\u1ec3 t\u1ea1o ra c\u00e1c m\u00f4 h\u00ecnh b\u1ec7nh trong ph\u00f2ng th\u00ed nghi\u1ec7m \u0111\u1ec3 nghi\u00ean c\u1ee9u c\u01a1 ch\u1ebf b\u1ec7nh v\u00e0 t\u00ecm ra nh\u1eefng ph\u01b0\u01a1ng ph\u00e1p m\u1edbi \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb.<\/div>\n
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2. \u0110i\u1ec1u tr\u1ecb b\u1ec7nh<\/div>\n
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M\u1ed9t trong nh\u1eefng \u1ee9ng d\u1ee5ng \u0111\u1ea7y h\u1ee9a h\u1eb9n c\u1ee7a CRISPR-Cas9 l\u00e0 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh. V\u1edbi kh\u1ea3 n\u0103ng ch\u1ec9nh s\u1eeda gen, ch\u00fang ta c\u00f3 th\u1ec3 s\u1eeda ch\u1eefa c\u00e1c l\u1ed7i gen g\u00e2y ra b\u1ec7nh v\u00e0 \u0111i\u1ec1u ch\u1ec9nh ho\u1ea1t \u0111\u1ed9ng c\u1ee7a gen \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb c\u00e1c b\u1ec7nh di truy\u1ec1n. V\u00ed d\u1ee5, CRISPR-Cas9 c\u00f3 th\u1ec3 \u0111\u01b0\u1ee3c s\u1eed d\u1ee5ng \u0111\u1ec3 lo\u1ea1i b\u1ecf gen g\u00e2y ung th\u01b0 ho\u1eb7c thay th\u1ebf c\u00e1c gen b\u1ecb h\u1ecfng trong b\u1ec7nh nh\u00e2n. \u0110i\u1ec1u n\u00e0y m\u1edf ra hy v\u1ecdng m\u1edbi cho vi\u1ec7c \u0111i\u1ec1u tr\u1ecb c\u00e1c b\u1ec7nh hi\u1ec3m ngh\u00e8o m\u00e0 tr\u01b0\u1edbc \u0111\u00e2y kh\u00f4ng c\u00f3 ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb hi\u1ec7u qu\u1ea3.<\/div>\n
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3. T\u1ea1o sinh v\u1eadt bi\u1ebfn \u0111\u1ed5i<\/div>\n
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M\u1ed9t \u1ee9ng d\u1ee5ng quan tr\u1ecdng kh\u00e1c c\u1ee7a CRISPR-Cas9 l\u00e0 t\u1ea1o sinh v\u1eadt bi\u1ebfn \u0111\u1ed5i. B\u1eb1ng c\u00e1ch ch\u1ec9nh s\u1eeda gen, ch\u00fang ta c\u00f3 th\u1ec3 t\u1ea1o ra c\u00e1c bi\u1ebfn th\u1ec3 sinh v\u1eadt v\u1edbi c\u00e1c \u0111\u1eb7c t\u00ednh mong mu\u1ed1n. V\u00ed d\u1ee5, ch\u00fang ta c\u00f3 th\u1ec3 t\u1ea1o ra c\u00e2y tr\u1ed3ng ch\u1ed1ng ch\u1ecbu b\u1ec7nh t\u1ed1t h\u01a1n ho\u1eb7c gia s\u00fac c\u00f3 kh\u1ea3 n\u0103ng ch\u1ed1ng l\u1ea1i c\u00e1c b\u1ec7nh truy\u1ec1n nhi\u1ec5m. \u0110i\u1ec1u n\u00e0y c\u00f3 th\u1ec3 gi\u00fap n\u00e2ng cao n\u0103ng su\u1ea5t n\u00f4ng nghi\u1ec7p v\u00e0 gi\u1ea3m s\u1eed d\u1ee5ng thu\u1ed1c tr\u1eeb s\u00e2u.<\/div>\n
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Ti\u1ec1m n\u0103ng c\u1ee7a CRISPR-Cas9 trong t\u1ea1o gi\u1ed1ng v\u1eadt nu\u00f4i<\/figcaption><\/figure>\n<\/div>\n
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Ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p ch\u1ec9nh s\u1eeda gen CRISPR<\/div>\n
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Nh\u1edd v\u00e0o ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p ch\u1ec9nh s\u1eeda gen CRISPR, ch\u00fang ta \u0111\u00e3 th\u1ea5y nh\u1eefng c\u1ea3i ti\u1ebfn \u0111\u00e1ng k\u1ec3 trong qu\u00e1 tr\u00ecnh ch\u1ec9nh s\u1eeda gen v\u00e0 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh. D\u01b0\u1edbi \u0111\u00e2y l\u00e0 m\u1ed9t s\u1ed1 ti\u1ebfn b\u1ed9 quan tr\u1ecdng:<\/div>\n
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1. CRISPR-Cas12a<\/div>\n
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Cas12a nh\u1eadn di\u1ec7n v\u00e0 c\u1eaft DNA ch\u00ednh x\u00e1c h\u01a1n, gi\u1ea3m nguy c\u01a1 g\u00e2y t\u1ed5n th\u01b0\u01a1ng \u0111\u1ebfn DNA kh\u00f4ng mong mu\u1ed1n.<\/figcaption><\/figure>\n<\/div>\n
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CRISPR-Cas12a l\u00e0 m\u1ed9t bi\u1ebfn th\u1ec3 m\u1edbi c\u1ee7a CRISPR-Cas9. So v\u1edbi Cas9, Cas12a c\u00f3 kh\u1ea3 n\u0103ng nh\u1eadn di\u1ec7n v\u00e0 c\u1eaft DNA ch\u00ednh x\u00e1c h\u01a1n. \u0110i\u1ec1u n\u00e0y l\u00e0m cho vi\u1ec7c ch\u1ec9nh s\u1eeda gen tr\u1edf n\u00ean d\u1ec5 d\u00e0ng h\u01a1n v\u00e0 gi\u00fap gi\u1ea3m nguy c\u01a1 g\u00e2y t\u1ed5n th\u01b0\u01a1ng \u0111\u1ebfn DNA kh\u00f4ng mong mu\u1ed1n.<\/div>\n
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2. CRISPR-Cas13<\/div>\n
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CRISPR-Cas13 l\u00e0 m\u1ed9t bi\u1ebfn th\u1ec3 m\u1edbi kh\u00e1c c\u1ee7a CRISPR-Cas9. Kh\u00e1c v\u1edbi Cas9 v\u00e0 Cas12a, Cas13 kh\u00f4ng nh\u1eafm m\u1ee5c ti\u00eau v\u00e0o DNA m\u00e0 nh\u1eafm v\u00e0o RNA. \u0110i\u1ec1u n\u00e0y cho ph\u00e9p ch\u00fang ta ch\u1ec9nh s\u1eeda v\u00e0 ki\u1ec3m so\u00e1t ho\u1ea1t \u0111\u1ed9ng c\u1ee7a RNA, m\u1edf ra nh\u1eefng c\u01a1 h\u1ed9i m\u1edbi trong nghi\u00ean c\u1ee9u v\u00e0 \u0111i\u1ec1u tr\u1ecb c\u00e1c b\u1ec7nh li\u00ean quan \u0111\u1ebfn RNA nh\u01b0 vi r\u00fat.<\/div>\n
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M\u1ee5c ti\u00eau c\u1ee7a Cas 13 l\u00e0 ARN kh\u00e1c v\u1edbi ADN nh\u01b0 Cas12<\/figcaption><\/figure>\n<\/div>\n
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3. CRISPR-Cas9 \u0111\u01b0\u1ee3c c\u1ea3i ti\u1ebfn<\/div>\n
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M\u1eb7c d\u00f9 CRISPR-Cas9 \u0111\u00e3 mang l\u1ea1i nhi\u1ec1u l\u1ee3i \u00edch cho nghi\u00ean c\u1ee9u v\u00e0 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh, nh\u01b0ng n\u00f3 v\u1eabn c\u00f2n m\u1ed9t s\u1ed1 h\u1ea1n ch\u1ebf. M\u1ed9t s\u1ed1 nh\u00e0 khoa h\u1ecdc \u0111\u00e3 c\u1ea3i ti\u1ebfn CRISPR-Cas9 \u0111\u1ec3 gi\u1ea3m nguy c\u01a1 g\u00e2y t\u1ed5n th\u01b0\u01a1ng \u0111\u1ebfn DNA kh\u00f4ng mong mu\u1ed1n v\u00e0 t\u0103ng t\u00ednh ch\u00ednh x\u00e1c c\u1ee7a qu\u00e1 tr\u00ecnh ch\u1ec9nh s\u1eeda gen. C\u00e1c c\u1ea3i ti\u1ebfn n\u00e0y bao g\u1ed3m vi\u1ec7c s\u1eed d\u1ee5ng protein Cas9 \u0111\u01b0\u1ee3c c\u1eaft th\u00e0nh hai ph\u1ea7n \u0111\u1ec3 gi\u1ea3m k\u00edch th\u01b0\u1edbc v\u00e0 t\u0103ng kh\u1ea3 n\u0103ng nh\u1eadn di\u1ec7n DNA, s\u1eed d\u1ee5ng Cas9 t\u1eeb vi khu\u1ea9n kh\u00e1c \u0111\u1ec3 tr\u00e1nh c\u00e1c v\u1ea5n \u0111\u1ec1 mi\u1ec5n d\u1ecbch, v\u00e0 s\u1eed d\u1ee5ng c\u00e1c lo\u1ea1i enzyme ch\u1ec9nh s\u1eeda gen kh\u00e1c nhau \u0111\u1ec3 t\u0103ng s\u1ef1 linh ho\u1ea1t.<\/div>\n
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Ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p ch\u1ec9nh s\u1eeda gen \u0111\u00e3 mang l\u1ea1i nh\u1eefng c\u1ea3i ti\u1ebfn \u0111\u00e1ng k\u1ec3 trong nghi\u00ean c\u1ee9u gen v\u00e0 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh. V\u1edbi kh\u1ea3 n\u0103ng ch\u1ec9nh s\u1eeda gen ch\u00ednh x\u00e1c v\u00e0 hi\u1ec7u qu\u1ea3, CRISPR-Cas9 \u0111\u00e3 m\u1edf ra nh\u1eefng c\u01a1 h\u1ed9i m\u1edbi trong vi\u1ec7c hi\u1ec3u r\u00f5 v\u1ec1 vai tr\u00f2 c\u1ee7a c\u00e1c gen v\u00e0 \u0111i\u1ec1u tr\u1ecb c\u00e1c b\u1ec7nh di truy\u1ec1n. Tuy nhi\u00ean, c\u00f2n r\u1ea5t nhi\u1ec1u c\u00f4ng vi\u1ec7c c\u1ea7n \u0111\u01b0\u1ee3c th\u1ef1c hi\u1ec7n \u0111\u1ec3 t\u1ed1i \u01b0u h\u00f3a qu\u00e1 tr\u00ecnh ch\u1ec9nh s\u1eeda gen v\u00e0 \u0111\u1ea3m b\u1ea3o an to\u00e0n cho con ng\u01b0\u1eddi. V\u1edbi s\u1ef1 ti\u1ebfp t\u1ee5c c\u1ee7a nghi\u00ean c\u1ee9u v\u00e0 ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p ch\u1ec9nh s\u1eeda gen, ch\u00fang ta c\u00f3 th\u1ec3 hy v\u1ecdng v\u00e0o t\u01b0\u01a1ng lai v\u1edbi nh\u1eefng ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb ti\u00ean ti\u1ebfn v\u00e0 m\u1ed9t cu\u1ed9c s\u1ed1ng kh\u1ecfe m\u1ea1nh h\u01a1n.<\/div>\n","protected":false},"excerpt":{"rendered":"

Trong nh\u1eefng n\u0103m g\u1ea7n \u0111\u00e2y, ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p ch\u1ec9nh s\u1eeda gen CRISPR \u0111\u00e3 t\u1ea1o ra m\u1ed9t cu\u1ed9c c\u00e1ch m\u1ea1ng trong l\u0129nh v\u1ef1c y h\u1ecdc v\u00e0 c\u00f4ng ngh\u1ec7 sinh h\u1ecdc. CRISPR-Cas9, h\u1ec7 th\u1ed1ng ch\u1ec9nh s\u1eeda gen d\u1ef1a tr\u00ean CRISPR, \u0111\u00e3 tr\u1edf th\u00e0nh c\u00f4ng c\u1ee5 quan tr\u1ecdng cho c\u00e1c nghi\u00ean c\u1ee9u gen v\u00e0 \u0111i\u1ec1u tr\u1ecb 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