Patents by Inventor Gustavo D. Aguirre
Gustavo D. Aguirre 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).
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Publication number: 20230139443Abstract: Methods for treating bestrophinopathies are provided herein. The method includes, administering to an eye of the subject a dose of a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid sequence encoding a human BEST1 protein, wherein the subject has at least one mutant BEST1 allele. Also provided are methods for evaluating treatments for retinal degeneration.Type: ApplicationFiled: February 28, 2021Publication date: May 4, 2023Inventors: Karina E. Guziewicz, Artur V. Cideciyan, William A. Beltran, Samuel G. Jacobson, Gustavo D. Aguirre
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Publication number: 20230112568Abstract: Methods for treating bestrophinopathies are provided herein. The method includes, administering to an eye of the subject a dose of a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid sequence encoding a human BEST1 protein, wherein the subject has two mutant BEST1 alleles. Also provided are methods for evaluating treatments for retinal degeneration.Type: ApplicationFiled: February 28, 2021Publication date: April 13, 2023Inventors: Karina E. Guziewicz, Artur V. Cideciyan, William A. Beltran, Samuel G. Jacobson, Gustavo D. Aguirre
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Publication number: 20210324387Abstract: Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.Type: ApplicationFiled: May 21, 2021Publication date: October 21, 2021Applicants: Univeristy of Florida Research Foundation, Incorporated, The Trustees of the University of PennsylvaniaInventors: Alfred S. Lewin, William W. Hauswirth, Michael T. Massengill, William Beltran, Gustavo D. Aguirre, Artur Cideciyan, Samuel Jacobson
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Patent number: 11118185Abstract: Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.Type: GrantFiled: March 1, 2017Date of Patent: September 14, 2021Assignees: University of Florida Research Foundation, Incorporated, The Trustees of the University of PennsylvaniaInventors: Alfred S. Lewin, William W. Hauswirth, Michael T. Massengill, William Beltran, Gustavo D. Aguirre, Artur Cideciyan, Samuel Jacobson
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Publication number: 20210207147Abstract: Aspects of the disclosure relate to methods and compositions useful for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering RNA to a subject in order to reduce expression of one or both alleles of an endogenous RHO gene (for example a mutant rho allele associated with retinitis pigmentosa) in a subject. In some embodiments, a replacement RHO coding sequence that is resistant to the interfering RNA also is delivered to the subject.Type: ApplicationFiled: June 3, 2019Publication date: July 8, 2021Applicants: University of Florida Research Foundation, Incorporated, The Trustees of the University of PennsylvaniaInventors: Alfred S. Lewin, William W. Hauswirth, Michael T. Massengill, William Beltran, Gustavo D. Aguirre, Artur Cideciyan, Samuel Jacobson
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Patent number: 10383922Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to the subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: GrantFiled: September 8, 2017Date of Patent: August 20, 2019Assignees: The Trustees of the University of Pennsylvania, University of Florida Research Foundation, IncorporatedInventors: William A Beltran, Gustavo D Aguirre, Samuel G Jacobson, Artur V Cideciyan, Alfred S Lewin, Sanford L Boye, William W Hauswirth, Wen-Tao Deng
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Publication number: 20190093111Abstract: Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.Type: ApplicationFiled: March 1, 2017Publication date: March 28, 2019Applicants: University of Florida Research Foundation, Incorporated, The Trustees of the University of PennsylvaniaInventors: Alfred S. Lewin, William W. Hauswirth, MIchael T. Massengill, William Beltran, Gustavo D. Aguirre, Artur Cideciyan, Samuel Jacovson
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Publication number: 20180036385Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: ApplicationFiled: September 8, 2017Publication date: February 8, 2018Inventors: William A. BELTRAN, Gustavo D. AGUIRRE, Samuel G. JACOBSON, Artur V. CIDECIYAN, Alfred S. LEWIN, Sanford L. BOYE, William W. HAUSWIRTH, Wen-Tao DENG
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Patent number: 9770491Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to a subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: GrantFiled: January 23, 2013Date of Patent: September 26, 2017Assignees: The Trustees of the University of Pennsylvania, Univeristy of Florida Research Foundation, IncorporatedInventors: William A Beltran, Gustavo D Aguirre, Samuel G Jacobson, Artur V Cideciyan, Alfred S Lewin, Sanford L Boye, William W Hauswirth, Wen-Tao Deng
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Publication number: 20170014529Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: ApplicationFiled: July 27, 2016Publication date: January 19, 2017Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William H. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Publication number: 20160263246Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: ApplicationFiled: May 4, 2016Publication date: September 15, 2016Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Patent number: 9433688Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: GrantFiled: June 20, 2014Date of Patent: September 6, 2016Assignees: The Trustees of the University of Pennsylvania, University of Florida Research Foundation, Incorporated, Cornell Research Foundation, Inc.Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Publication number: 20150202269Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: ApplicationFiled: January 23, 2013Publication date: July 23, 2015Inventors: William A Beltran, Gustavo D Aguirre, Samuel G Jacobson, Artur V Cideciyan, Alfred S Lewin, Sanford L Boye, William W Hauswirth, Wen-Tao Deng
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Publication number: 20140377224Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: ApplicationFiled: June 20, 2014Publication date: December 25, 2014Applicants: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA, UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED, CORNELL RESEARCH FOUNDATION, INC.Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Publication number: 20120225930Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: ApplicationFiled: February 28, 2012Publication date: September 6, 2012Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Patent number: 8147823Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: GrantFiled: July 8, 2010Date of Patent: April 3, 2012Assignees: The Trustees of the University of Pennsylvania, University of Florida Research Foundation, Incorporated, Cornell Research Foundation Inc.Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Publication number: 20100272688Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: ApplicationFiled: July 8, 2010Publication date: October 28, 2010Applicants: The Trustees of the University of Pennsylvania, University of Florida Research Foundation, Incorporated, Cornell Research Fountaintion, Inc.Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Patent number: 7811761Abstract: Provided is a method for identifying dogs as likely to be genetically normal, carriers of, or affected with progressive rod-cone degeneration comprising analyzing nucleic acids from a dog and determining the presence or absence of one or more prcd polymorphisms in the nucleic acids. Representative prcd polymorphisms characteristic of a prcd haplotype are also provided.Type: GrantFiled: December 13, 2006Date of Patent: October 12, 2010Assignee: Cornell Research Foundation, Inc.Inventors: Gregory M. Acland, Gustavo D. Aguirre, Orly Goldstein, Barbara Zangerl, Susan Pearce-Kelling, Duska J. Sidjanin, Jeanette S. Felix
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Patent number: 7723507Abstract: The invention relates to a method for identifying dogs which are genetically normal, heterozygous for, or homozygous for the mutation primarily responsible for Collie eye anomaly (CEA). The method comprises the steps of obtaining a biological sample from a dog and testing DNA in the biological sample for the presence or absence of a 7.8 kilobase deletion within chromosome 37 in which the CEA mutation is located. No deletion is indicative of a normal dog. A deletion on one allele of chromosome 37 is indicative of a dog that is heterozygous for the CEA mutation. A deletion in both alleles of chromosome 37 are indicative of a dog that is homozygous for the CEA mutation. Also provided is a kit for identifying a dog as normal, heterozygous for, or homozygous for the CEA mutation.Type: GrantFiled: September 22, 2008Date of Patent: May 25, 2010Assignee: Cornell Research Foundation, Inc.Inventors: Gregory M. Acland, Anna V. Kukekova, Gustavo D. Aguirre, Elaine Ostrander, Dayna Akey, Orly Goldstein
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Publication number: 20090176225Abstract: The invention relates to a method for identifying dogs which are genetically normal, heterozygous for, or homozygous for the mutation primarily responsible for Collie eye anomaly (CEA). The method comprises the steps of obtaining a biological sample from a dog and testing DNA in the biological sample for the presence or absence of a 7.8 kilobase deletion within chromosome 37 in which the CEA mutation is located. No deletion is indicative of a normal dog. A deletion on one allele of chromosome 37 is indicative of a dog that is heterozygous for the CEA mutation. A deletion in both alleles of chromosome 37 are indicative of a dog that is homozygous for the CEA mutation. Also provided is a kit for identifying a dog as normal, heterozygous for, or homozygous for the CEA mutation.Type: ApplicationFiled: September 22, 2008Publication date: July 9, 2009Inventors: Gregory M. Acland, Anna V. Kukekova, Gustavo D. Aguirre, Elaine Ostrander, Dayna Akey, Orly Goldstein