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Updated: May 5, 2026

Transfection, Selection, and Colony-picking of Human Induced Pluripotent Stem Cells TALEN-targeted with a GFP Gene into the AAVS1 Safe Harbor

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Published on: February 1, 2015

"セーフハーバー"遺伝子療法のアプローチは,最初の成功を収めるかもしれない

Science (New York, N.Y.)

|January 17, 2025

まとめ

新種の遺伝子エディタが肝臓の重要な酵素を重度の代謝障害を患っている乳児に回復させましたこの発見は稀な遺伝疾患の治療に新しい希望をもたらします

科学分野:

生物化学
遺伝学
小児科

背景:

特定の代謝状態の乳児には肝臓の酵素が欠けていて,深刻な健康問題が生じます.
現在,このような代謝障害の治療は限られており,根本的な酵素欠乏症を完全に解決できない可能性があります.

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Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications

Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications

Published on: February 9, 2024

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Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...