Haemophilia is a serious bleeding disorder caused by blood deficiency of clotting factors (factor VIII and factor IX proteins). It can lead to spontaneous bleeding episodes, joint damage, and a reduced quality of life for patients and their families.
The new gene therapy has shown promising results during the first phase trial on five patients, aged between 22 and 41. The annualised bleeding rate has been observed to be zero in all five patients over a cumulative follow-up of 81 months.
Supported by the Department of Biotechnology, this first-in-human gene therapy for haemophilia-A patients, has been developed by Centre for Stem Cell Research (CSCR) at CMC, Vellore. Results of the study conducted on the patients have been published in the recent edition of New England Journal of Medicine.
この記事は The New Indian Express Mysuru の December 17, 2024 版に掲載されています。
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この記事は The New Indian Express Mysuru の December 17, 2024 版に掲載されています。
7 日間の Magzter GOLD 無料トライアルを開始して、何千もの厳選されたプレミアム ストーリー、9,000 以上の雑誌や新聞にアクセスしてください。
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