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Compositions For Enhancing Transport Of Molecules Into Cells
专利权人:
SAREPTA THERAPEUTICS; INC.
发明人:
Patrick L. Iversen,Hong M. Moulton,Michelle H. Nelson,David A. Stein,Andrew D. Kroeker
申请号:
US16379046
公开号:
US20200016280A1
申请日:
2019.04.09
申请国别(地区):
US
年份:
2020
代理人:
摘要:
Compositions and methods for enhancing delivery of molecules, e.g. biological agents, into cells are described. The composition is a conjugate of the biological agent, preferably a nucleic acid analog having a substantially uncharged backbone, covalently linked to a peptide transporter moiety as described. Conjugation of the peptide transporter to a substantially uncharged nucleic acid analog, such as a morpholino oligomer, is also shown to enhance binding of the oligomer to its target sequence and enhance antisense activity.
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