Patents by Inventor Stuart H. Orkin

Stuart H. Orkin has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • Modulation of BCL11A for treatment of hemoglobinopathies
    Patent number: 9228185
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Grant
    Filed: January 17, 2013
    Date of Patent: January 5, 2016
    Assignees: CHILDREN'S MEDICAL CENTER CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Stuart H. Orkin, Vijay G. Sankaran
  • TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION
    Publication number: 20150307867
    Abstract: Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.
    Type: Application
    Filed: November 27, 2013
    Publication date: October 29, 2015
    Applicant: CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Stuart H. ORKIN, Daniel E. BAUER, Jian XU
  • NUCLEASE-MEDIATED REGULATION OF GENE EXPRESSION
    Publication number: 20150132269
    Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.
    Type: Application
    Filed: November 13, 2014
    Publication date: May 14, 2015
    Inventors: Stuart H. Orkin, Andreas Reik, Fyodor Urnov
  • COMBINATORIAL COMPOSITIONS AND METHODS OF TREATING HEMOGLOBINOPATHIES
    Publication number: 20140271657
    Abstract: Provided herein are compositions and methods for increasing fetal hemoglobin expression (HbF) for the treatment and/or amelioration of the symptoms of a hemoglobinpathy in a mammal. The compositions comprise a combination of one or more inhibitors of BCL11A expression or activity with epigenetic modifiers, such as inhibitors of DNA methylation and/or one or more inhibitors of histone deacetylases.
    Type: Application
    Filed: October 12, 2012
    Publication date: September 18, 2014
    Applicant: CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Stuart H. Orkin, Jian Xu, Peng Cong
  • DNA encoding Von Willebrand Factor (VWF) and methods and cells for producing VFW, and VFW produced by the DNA, methods and cells
    Patent number: 8597910
    Abstract: Von Willebrand's Factor (VWF) is produced using an expression vector that includes: 1) a DNA sequence encoding a functional VWF protein; and 2) regulatory DNA capable of effecting expression of that DNA sequence in a host cell transformed with the vector. Restriction fragment length polymorphisms (RFLP's) associated with the VWF gene are identified and used in a probe for determining the source of a VWF gene in a DNA sample. The gene for VWF is localized to the short arm of human chromosome 12 (12p).
    Type: Grant
    Filed: July 23, 1990
    Date of Patent: December 3, 2013
    Assignee: Children's Medical Center Corporation
    Inventors: David Ginsburg, Stuart H. Orkin, Randal J. Kaufman
  • MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
    Publication number: 20130129629
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Application
    Filed: January 17, 2013
    Publication date: May 23, 2013
    Applicants: PRESIDENT AND FELLOWS OF HARVARD COLLEGE, CHILDREN'S MEDICAL CENTER CORPORATION
    Inventors: Stuart H. Orkin, Vijay G. Sankaran
  • Modulation of BCL11A for treatment of hemoglobinopathies
    Patent number: 8383604
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Grant
    Filed: September 14, 2009
    Date of Patent: February 26, 2013
    Assignees: Children's Medical Center Corporation, President and Fellows of Harvard College
    Inventors: Stuart H. Orkin, Vijay G. Sankaran
  • MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
    Publication number: 20110182867
    Abstract: The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
    Type: Application
    Filed: September 14, 2009
    Publication date: July 28, 2011
    Applicants: CHILDREN'S MEDICAL CENTER CORPORATION, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
    Inventors: Stuart H. Orkin, Vijay G. Sankaran