序号 专利名 申请号 申请日 公开(公告)号 公开(公告)日 发明人
1 METHOD US14598948 2015-01-16 US20160206704A1 2016-07-21 Robert MacLaren
A method of using a vector comprising a transgene to treat a disease or condition of the eye, the method comprising the steps: (a) administering a solution to a mammalian subject by subretinal injection in an amount effective to at least partially detach the retina to form a subretinal bleb, wherein the solution does not comprise the vector; and (b) administering a medicament composition by subretinal injection into the bleb formed by step (a), wherein the medicament comprises the vector and is injected in an amount effective to treat the disease or condition; wherein the transgene is expressible in cells of the mammalian subject.
2 Modulators of RabGGT and methods of use thereof US10638225 2003-08-07 US20040142888A1 2004-07-22 Veeraswamy Manne; Mark Lynch; Petra B. Ross-MacDonald; Terry Stouch; Naomi Laing; Pamela Carroll; Kevin Fitzgerald; Louis J. Lombardo; Michael R. Costa; Mark E. Maxwell; Rachel M. Kindt; Mark R. Lackner; Tak Hung; Carol L. O'Brian; Hai Guang Zhang; Katherine S. Brown; Jae Moon Lee
The present invention provides methods for inducing apoptosis in a cell, the methods generally involving contacting the cell with an agent that reduces the level and/or activity of RabGGT. The present invention further provides methods for treating a disorder related to unwanted cell proliferation in an individual, the methods generally involving administering to the individual an agent that reduces the level and/or activity of RabGGT. The present invention further provides methods for reducing apoptosis in a cell, the methods generally involving increasing the level and/or activity of RabGGT in the cell. The present invention further provides methods for treating disorders associated with excessive apoptosis. The present invention further provides methods for identifying a cell that is amenable to treatment with the methods of the present invention. The present invention further provides methods for modulating a binding event between RabGGT and a RabGGT interacting protein. The present invention further provides a 3-dimensional structure of RabGGT, and methods of use of the structure to identify compounds that modulate RabGGT activity.
3 RAB ESCORT PROTEIN POTENCY ASSAY US15806136 2017-11-07 US20180135097A1 2018-05-17 John Fraser WRIGHT; Marina SUMAROKA
Methods for measuring REP-1 and REP-2 activity are provided. In certain embodiments, a method includes: (a) contacting cells that do not express endogenous functional REP-1 or REP-2 protein with an adeno-associated viral (AAV) vector comprising a CHM gene encoding a REP-1 protein or CHM like gene encoding a REP-2 protein under conditions allowing cell transduction; (b) incubating transduced cells under conditions allowing expression of the encoded REP-1 or REP-2 protein; (c) lysing the transduced cells to produce an extract comprising the encoded REP-1 or REP-2 protein and Rab small GTPase (Rabs); (d) incubating said extract with a Rab substrate for a period of time and under conditions allowing prenylation of the Rab thereby forming prenylated Rab; and (e) detecting and/or quantifying the prenylated Rab, wherein the amount of prenylated Rab reflects REP-1 or REP-2 activity thereby measuring REP-1 or REP-2 activity.
4 MODULATORS OF RABGGT AND METHODS OF USE THEREOF EP03785122 2003-08-07 EP1534862A4 2007-10-10 MANNE VEERASWAMY; LYNCH MARK; ROSS-MACDONALD PETRA B; STOUCH TERRY; LANGE NAOMI; CARROLL PAMELA; FITZGERALD KEVIN; COSTA MICHAEL R; MAXWELL MARK E; KINDT RACHEL M; LACKNER MARK R; HUNG TAK; O'BRIEN CAROL L; ZHANG HAI GUANG; BROWN KATHERINE S; LEE JAE MOON; LOMBARDO LOUIS J
The present invention provides methods for inducing apoptosis in a cell, the methods generally involving contacting the cell with an agent that reduces the level and/or activity of RabGGT. The present invention further provides methods for treating a disorder related to unwanted cell proliferation in an individual, the methods generally involving administering to the individual an agent that reduces the level and/or activity of RabGGT. The present invention further provides methods for reducing apoptosis in a cell, the methods generally involving increasing the level and/or activity of RabGGT in the cell. The present invention further provides methods for treating disorders associated with excessive apoptosis. The present invention further provides methods for identifying a cell that is amenable to treatment with the methods of the present invention. The present invention further provides methods for modulating a binding event between RabGGT and a RabGGT interacting protein. The present invention further provides a 3-dimensional structure of RabGGT, and methods of use of the structure to identify compounds that modulate RabGGT activity.
5 MODULATORS OF RABGGT AND METHODS OF USE THEREOF EP03785122.7 2003-08-07 EP1534862A2 2005-06-01 MANNE, Veeraswamy; LYNCH, Mark; ROSS-MACDONALD, Petra B.; STOUCH, Terry; LANGE, Naomi; CARROLL, Pamela; FITZGERALD, Kevin; COSTA, Michael, R.; MAXWELL, Mark E.; KINDT, Rachel M.; LACKNER, Mark R.; HUNG, Tak; O'BRIEN, Carol, L.; ZHANG, Hai Guang; BROWN, Katherine, S.; LEE, Jae Moon; LOMBARDO, Louis J.
The present invention provides methods for inducing apoptosis in a cell, the methods generally involving contacting the cell with an agent that reduces the level and/or activity of RabGGT. The present invention further provides methods for treating a disorder related to unwanted cell proliferation in an individual, the methods generally involving administering to the individual an agent that reduces the level and/or activity of RabGGT. The present invention further provides methods for reducing apoptosis in a cell, the methods generally involving increasing the level and/or activity of RabGGT in the cell. The present invention further provides methods for treating disorders associated with excessive apoptosis. The present invention further provides methods for identifying a cell that is amenable to treatment with the methods of the present invention. The present invention further provides methods for modulating a binding event between RabGGT and a RabGGT interacting protein. The present invention further provides a 3-dimensional structure of RabGGT, and methods of use of the structure to identify compounds that modulate RabGGT activity.
6 MODULATORS OF RABGGT AND METHODS OF USE THEREOF PCT/US0325001 2003-08-07 WO2004015130A3 2004-10-14 MANNE VEERASWAMY; LYNCH MARK; ROSS-MACDONALD PETRA B; STOUCH TERRY; LAING NAOMI; CARROLL PAMELA; FITZGERALD KEVIN; COSTA MICHAEL R; MAXWELL MARK E; KINDT RACHEL M; LACKNER MARK R; HUNG TAK; O'BRIEN CAROL L; ZHANG HAI GUANG; BROWN KATHERINE S; LEE JAE MOON; LOMBARDO LOUIS J
The present invention provides methods for inducing apoptosis in a cell, the methods generally involving contacting the cell with an agent that reduces the level and/or activity of RabGGT. The present invention further provides methods for treating a disorder related to unwanted cell proliferation in an individual, the methods generally involving administering to the individual an agent that reduces the level and/or activity of RabGGT. The present invention further provides methods for reducing apoptosis in a cell, the methods generally involving increasing the level and/or activity of RabGGT in the cell. The present invention further provides methods for treating disorders associated with excessive apoptosis. The present invention further provides methods for identifying a cell that is amenable to treatment with the methods of the present invention. The present invention further provides methods for modulating a binding event between RabGGT and a RabGGT interacting protein. The present invention further provides a 3-dimensional structure of RabGGT, and methods of use of the structure to identify compounds that modulate RabGGT activity.
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