公开(公告)号：WO2017136536A1

公开(公告)日：2017-08-10

申请号：PCT/US2017/016194

申请日：2017-02-02

申请人： UNIVERSITY OF MASSACHUSETTS

发明人： ESTEVES, Miguel Sena ,  BATISTA, Ana Rita ,  CHOUDHURY, Sourav Roy

IPC分类号： A61K38/10 ,  A61K38/16 ,  C12N15/63 ,  C12N15/79 ,  C12N15/86 ,  C12N15/864 ,  C12N15/867 ,  C12N15/869 ,  C12N15/64 ,  C12N5/10

CPC分类号： A61K38/1709 ,  A61K9/0085 ,  A61K31/4706 ,  A61K47/62 ,  A61K47/6455 ,  A61K48/00 ,  C07K5/0808 ,  C07K2319/00 ,  C12N15/63 ,  C12N15/64 ,  C12N15/79 ,  C12N15/86 ,  C12N15/864 ,  C12N15/867 ,  C12N15/869 ,  C12N2750/14132

摘要： The disclosure relates, in some aspects, to compositions and methods for enhanced delivery of a transgene to the central nervous system (CNS) of a subject. In some embodiments, the transgene is delivered by recombinant AAV (rAAV). In some embodiments, the method of enhancing transgene delivery comprises administering a blood brain barrier (BBB)-crossing molecule (e.g., K16ApoE) and an rAAV comprising a transgene to a subject.

摘要翻译： 本公开在一些方面涉及用于增强递送递送至受试者中枢神经系统（CNS）的转基因的组合物和方法。 在一些实施方案中，转基因通过重组AAV（rAAV）递送。 在一些实施方案中，增强转基因递送的方法包括向受试者施用血脑屏障（BBB） - 分子（例如K16ApoE）和包含转基因的rAAV。

公开(公告)号：WO2022066849A1

公开(公告)日：2022-03-31

申请号：PCT/US2021/051667

申请日：2021-09-23

申请人： UNIVERSITY OF MASSACHUSETTS

发明人： ESTEVES, Miguel Sena

IPC分类号： C07K14/47 ,  C12N15/00 ,  C12N15/09 ,  C12N15/12 ,  C12N15/63 ,  C12N15/86

摘要： Aspects of the disclosure relate to compositions and methods of treating certain genetic disease (e.g., Neurofibromatosis type I) by delivering functional neurofibromin 1(NF1) protein (e.g., mini-NFl protein and/or full-length NF1 protein) to target cell (e.g., cells and/or tissue of a subject). The disclosure is based, in part, on isolated nucleic acids (e.g., rAAV vectors) and rAAVs engineered to express a functional NF1 protein (e.g., mini-NFl protein and/or full-length NF1 protein) or variants thereof.