Patents Assigned to Children's Medical Research Institute
-
Publication number: 20240067678Abstract: The present disclosure relates generally to adeno-associated vims (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells. The disclosure includes AAV capsid polypeptides comprising a peptide modification in variable region 8 (VR VIII), where the peptide modification comprises a 7 amino acid insertion.Type: ApplicationFiled: December 16, 2021Publication date: February 29, 2024Applicant: Children's Medical Research InstituteInventors: Adrian WESTHAUS, Leszek LISOWSKI, Marti CABANES CREUS
-
Patent number: 11351272Abstract: Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provide are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells.Type: GrantFiled: June 24, 2019Date of Patent: June 7, 2022Assignees: THE SYDNEYCHILDREN'S HOSPITALS NETWORK (RANDWICK AND WESTMEAD) (INCORPORATING THE ROYAL ALEXANDRA HOSPITAL FOR CHILDREN), CHILDREN'S MEDICAL RESEARCH INSTITUTE, MOUNT SINAI HOSPITALInventors: Ian Alexander, Sharon Cunningham, Andras Nagy
-
Publication number: 20210388343Abstract: The present disclosure relates generally to nucleic acid molecules and methods for identifying AAV vectors with desirable properties, including nucleic acid molecules and methods useful for identifying novel cap genes for vectorization, production of AAV vectors and AAV libraries.Type: ApplicationFiled: October 17, 2019Publication date: December 16, 2021Applicant: Children's Medical Research InstituteInventors: Leszek LISOWSKI, Marti CABANES-CREUS, Adrian WESTHAUS
-
Patent number: 11104917Abstract: The present disclosure relates generally to promoters derived from the AAV anti-sense strand, their use in the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the promoters. The present disclosure also relates to enhancers derived from the AAV anti-sense strand, their use in increasing the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the enhancers.Type: GrantFiled: May 4, 2016Date of Patent: August 31, 2021Assignees: CHILDREN'S MEDICAL RESEARCH INSTITUTE, THE SYDNEY CHILDREN'S HOSPITALS NETWORK (RANDWICK AND WESTMEAD) (INCORPORATING THE ROYAL ALEXANDRA HOSPITAL FOR CHILDREN)Inventors: Grant Logan, Ian Alexander, Allison Dane
-
Publication number: 20210122789Abstract: The present disclosure relates generally to polypeptides derived from marsupial adeno-associated vims (AAV). The disclosure is also related to nucleic acid molecules encoding the polypeptides, and vectors comprising the nucleic acid molecules, and AAV vectors comprising the polypeptides. The disclosure also relates to uses of the nucleic acid molecules, polypeptides and AAV vectors, such as for capsid diversification.Type: ApplicationFiled: May 31, 2019Publication date: April 29, 2021Applicants: Children's Medical Research Institute, The Sydney Children's Hospitals Network (Randwick and Westmead) (Incorporating the Royal AlexandraInventors: Claus HALLWIRTH, Ian ALEXANDER
-
Patent number: 10882886Abstract: Polypeptides are encoded by an adeno-associated virus (AAV)-derived endogenous viral element (mAAV-EVE1) found within the germline of numerous closely-related marsupial species. Nucleic acid molecules encode the polypeptides. Vectors can include the nucleic acid molecules, and recombinant AAV virions can include the polypeptides. A chimeric capsid protein can also include an MAAV-EVE1 polypeptide.Type: GrantFiled: May 3, 2017Date of Patent: January 5, 2021Assignees: CHILDREN'S MEDICAL RESEARCH INSTITUTE, THE SYDNEY CHILDREN'S HOSPITALS NETWORK (RANDWICK AND WESTMEAD) (INCORPORATING THE ROYAL ALEXANDRA HOSPITAL FOR CHILDREN), THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES (WASHINGTON DC)Inventors: Claus Hallwirth, Ian Alexander, Richard Smith, Robert Kotin
-
Publication number: 20200385789Abstract: This invention relates generally to methods for assessing telomeres, including methods of measuring the length of a telomere and methods for detecting extension of a telomere and methods for measuring telomere extension, and associated systems.Type: ApplicationFiled: August 31, 2018Publication date: December 10, 2020Applicant: Children's Medical Research InstituteInventors: Hilda PICKETT, Joshua ALLEN
-
Publication number: 20190365926Abstract: Polynucleotides and vectors can be used for the expression of a transgene in cells, such as liver cells. The expression of the transgene from the polynucleotides and vectors can be useful in gene therapy. Various methods can be used for expressing the transgene from the polynucleotides and vectors in liver cells.Type: ApplicationFiled: January 10, 2018Publication date: December 5, 2019Applicants: Children's Medical Research Institute, The Sydney Children's Hospitals Network (Randwick and Westmead) (incorporating The Royal AlexandraInventors: Ian ALEXANDER, Sharon CUNNINGHAM
-
Publication number: 20190135870Abstract: Polypeptides are encoded by an adeno-associated virus (AAV)-derived endogenous viral element (mAAV-EVE1) found within the germline of numerous closely-related marsupial species. Nucleic acid molecules encode the polypeptides. Vectors can include the nucleic acid molecules, and recombinant AAV virions can include the polypeptides. A chimeric capsid protein can also include an MAAV-EVE1 polypeptide.Type: ApplicationFiled: May 3, 2017Publication date: May 9, 2019Applicants: Children's Medical Research Institute, The Sydney Children's Hospitals Network (Randwick and Westmead) (incorporating The Royal Alexanndra, The United States of America, as represented by the Secretary, Department of Health and HumanInventors: Claus Hallwirth, Ian Alexander, Richard Smith, Robert Kotin
-
Methods and assays for the detection of alternative lengthening of telomeres (ALT) activity in cells
Patent number: 8999643Abstract: The invention relates to methods and assays for the detection of active Alternative Lengthening of Telomeres (ALT) activity in cells. The methods and assays involve detecting or assaying for partially double-stranded telomeric circles wherein the presence of said circles is specific for cells comprising an active ALT mechanism. In some embodiments the methods find application in, inter alia, determining the level of ALT activity in a cell, determining the ALT status of a cancer in a subject, diagnosing and/or treating disease, determining disease status, analysis of treatment efficacy, and the identification of novel therapeutic agents.Type: GrantFiled: September 21, 2010Date of Patent: April 7, 2015Assignee: Children's Medical Research InstituteInventors: Roger Robert Reddel, Jeremy David Henson -
Patent number: 8809386Abstract: There is provided a method for promoting dynamin ring formation and/or maintenance of dynamin rings in a cell, comprising treating the cell with an effective amount of a dynamin ring stabilizer, or a prodrug or pharmaceutically acceptable salt of the dynamin ring stabilizer. The maintenance or accumulation of dynamin ring formation has particular application in the prophylaxis or treatment of a kidney disease or condition characterized by proteinuria. A dynamin ring stabilizer can be any agent that interacts with dynamin to promote dynamin ring assembly and/or inhibit dynamin ring disassembly. There are also provided methods for prophylaxis or treatment of podocyte dysfunction and/or maintaining or inducing actin cytoskeleton formation in a cell utilizing dynamin ring stabilizers, and for screening a test agent for use as a dynamin ring stabilizer.Type: GrantFiled: May 21, 2010Date of Patent: August 19, 2014Assignees: Children's Medical Research Institute, The General Hospital CorporationInventors: Phillip J. Robinson, Sanja Sever
-
Publication number: 20120122968Abstract: There is provided a method for promoting dynamin ring formation and/or maintenance of dynamin rings in a cell, comprising treating the cell with an effective amount of a dynamin ring stabilizer, or a prodrug or pharmaceutically acceptable salt of the dynamin ring stabilizer. The maintenance or accumulation of dynamin ring formation has particular application in the prophylaxis or treatment of a kidney disease or condition characterized by proteinuria. A dynamin ring stabilizer can be any agent that interacts with dynamin to promote dynamin ring assembly and/or inhibit dynamin ring disassembly. There are also provided methods for prophylaxis or treatment of podocyte dysfunction and/or maintaining or inducing actin cytoskeleton formation in a cell utilizing dynamin ring stabilizers, and for screening a test agent for use as a dynamin ring stabilizer.Type: ApplicationFiled: May 21, 2010Publication date: May 17, 2012Applicants: THE GENERAL HOSPITAL CORPORATION, CHILDREN'S MEDICAL RESEARCH INSTITUTEInventors: Phillip J. Robinson, Sanja Sever
-
Publication number: 20080306007Abstract: Inhibitors of syndapin I binding to dynamin I (DynI) are provided. Examples include mimetics of a region of DynI including the serine residues S774 and S778 or phosphorylatable amino acids in homologous positions. Typically, the mimetics exclude or do not imitate at least one phosphorylation site provided by the serine residues or phosphorylatable amino acids. Peptide fragment inhibitors comprising or consisting of this region of DynI are also described. The inhibitors have application in the prophylaxis or treatment of neurological diseases or conditions. The inhibitors can also be used to inhibit neuronal cell vesicle trafficking and synaptic signal transmission.Type: ApplicationFiled: April 30, 2008Publication date: December 11, 2008Applicants: Newcastle Innovation Limited, Children's Medical Research Institute, The University Court of The University of EdinburghInventors: Adam McCluskey, Phillip Robinson, Michael Cousin