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THERAPEUTIC CELL SYSTEMS AND METHODS FOR TREATING HOMOCYSTINURIA
专利权人:
Rubius Therapeutics; Inc.
发明人:
Lenka Hoffman,Thomas Joseph Wickham
申请号:
US16359978
公开号:
US20190309271A1
申请日:
2019.03.20
申请国别(地区):
US
年份:
2019
代理人:
摘要:
The present disclosure relates to erythroid cells that have been engineered to express a homocysteine reducing polypeptide, or a variant thereof, or a homocysteine degrading polypeptide, or a variant thereof. The engineered erythroid cells may further comprise an amino acid transporter, for example a homocysteine transporter or a serine transporter, or a cystathionine degrading polypeptide. The engineered erythroid cells of the present disclosure are useful in reducing the level of homocysteine in a subject. The engineered erythroid cells of the present disclosure are further useful in methods of treating homocystinuria.
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