The Gene Therapy
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Abstract
การรักษาโรคด้วยยีนคือการนำยีนปรกติเข้าสู่ร่างกายเพื่อแก้ไข ทดแทนหรือทำลายยีนที่ผิดปรกติที่เป็นสาเหตุ ของการเกิดโรค เช่นโรคทางพันธุกรรมและโรคติดเชื้อบางชนิด โดยมีองค์ประกอบของการรักษาคือ ยีนเพื่อการรักษา พาหะ และเซลล์เป้าหมาย หัวใจหลักของความสำเร็จของการรักษาโรคด้วยวิธีนี้อยู่ที่ความสามารถในการนำส่งยีน เพื่อการรักษาเข้าสู่เซลล์เป้าหมายซึ่งแบ่งออกเป็นสองวิธีคือ การไม่ใช้เซลล์เป็นพาหะร่วมและการใช้เซลล์เป็นพาหะ ร่วมที่เรียกว่า "genetic modified cell" ซึ่งถือว่าเป็นการผนวกกำลังการรักษาระหว่างการรักษาโรคด้วยยีนและการ รักษาโรคด้วยเซลล์ การรักษาโรคด้วยยีนมีหลายรูปแบบโดยแบ่งเป็นสามหลักการคือ การนำยีนปรกติเข้าร่างกายเพื่อ ทำหน้าที่แทน ทำลาย และเข้าจับกับโปรตีนที่ใช้ในการควบคุมการแสดงออกของยีนที่ผิดปรกตินั้นโดยมีรูปแบบย่อย ดังนี้ 1) การใช้พลาสมิด 2) แอนตี้ยีนและแอนตี้เซนส์ โอลิโกนิวคลีโอไทด์3) ไรโบไซม์ 4) ดีเอนเอไซม์ 5) ดีคอยด์ 6) แอปทาเมอร์ และ 7) อาร์เอนเอไอ การรักษาโรคด้วยยีนถูกนำมาศึกษาวิจัยในมนุษย์ตั้งแต่ ค.ศ. 1990 และปัจจุบันนี้ได้รับการอนุมัติโครงการประมาณ 100 โครงการทั่วโลกซึ่งโดยส่วนใหญ่ศึกษาวิจัยอยู่ในระยะที่ 1 ในโรคมะเร็งและโรคหัวใจและหลอดเลือด ตามลำดับ
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