Patents by Inventor Matthias HEIDENREICH
Matthias HEIDENREICH 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: 20240124860Abstract: In one aspect, embodiments disclosed herein are directed to engineered CRISPRCas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector protein is a Type II effector protein. In certain other example embodiments, the Type V effector protein is Cas9 or an orthologs or engineered variant thereof. Example Cas9 proteins suitable for use in the embodiments disclosed herein are discussed in further detail below.Type: ApplicationFiled: October 17, 2023Publication date: April 18, 2024Inventors: Feng Zhang, David Arthur Scott, Winston Xia Yan, Sourav Choudhury, Matthias Heidenreich
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Publication number: 20230108784Abstract: Embodiments disclosed herein are directed to engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector protein is a Type V effector protein. In certain other example embodiments, the Type V effector protein is Cpf1. Embodiments disclosed herein are directed to viral vectors for delivery of CRISPR-Cas effector proteins, including Cpf1. In certain example embodiments, the vectors are designed so as to allow packaging of the CRISPR-Cas effector protein within a single vector. There is also an increased interest in the design of compact promoters for packing and thus expressing larger transgenes for targeted delivery and tissue-specificity.Type: ApplicationFiled: May 11, 2022Publication date: April 6, 2023Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Feng Zhang, David Arthur Scott, Winston Xia Yan, Sourav Choudhury, Matthias Heidenreich
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Publication number: 20230029506Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: ApplicationFiled: June 3, 2022Publication date: February 2, 2023Applicants: The Broad Institute, Inc., Massachusetts Institute of TechnologyInventors: Le CONG, David Benjamin Turitz COX, Matthias HEIDENREICH, Randall Jeffrey PLATT, Lukasz SWIECH, Feng ZHANG
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Patent number: 11407985Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: GrantFiled: June 10, 2016Date of Patent: August 9, 2022Assignees: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Le Cong, David Benjamin Turitz Cox, Matthias Heidenreich, Randall Jeffrey Platt, Lukasz Swiech, Feng Zhang
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Publication number: 20220186216Abstract: Compositions and methods for treating excising trinucleotide repeats, as well as for treating diseases and disorders associated with trinucleotide repeats are encompassed.Type: ApplicationFiled: February 25, 2022Publication date: June 16, 2022Applicant: Vertex Pharmaceuticals IncorporatedInventors: Gregoriy Aleksandrovich Dokshin, Matthias Heidenreich, Norzehan Abdul-Manan, Lu Gan, Jianming Liu, Guoxiang Ruan, Jesper Gromada, John Patrick Leonard, Zachary Michael Detwiler, Peter Thomas Hallock, David Esopi, Giselle Dominguez Gutierrez
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Patent number: 11352647Abstract: Embodiments disclosed herein are directed to engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector protein is a Type V effector protein. In certain other example embodiments, the Type V effector protein is Cpf1. Embodiments disclosed herein are directed to viral vectors for delivery of CRISPR-Cas effector proteins, including Cpf1. In certain example embodiments, the vectors are designed so as to allow packaging of the CRISPR-Cas effector protein within a single vector. There is also an increased interest in the design of compact promoters for packing and thus expressing larger transgenes for targeted delivery and tissue-specificity.Type: GrantFiled: August 17, 2017Date of Patent: June 7, 2022Assignees: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Feng Zhang, David Arthur Scott, Winston Xia Yan, Sourav Choudhury, Matthias Heidenreich
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Publication number: 20210361779Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: ApplicationFiled: March 15, 2021Publication date: November 25, 2021Applicants: The Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard CollegeInventors: Feng Zhang, Le Cong, Fei Ran, Matthias Heidenreich, Lukasz Swiech
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Patent number: 10946108Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: GrantFiled: December 16, 2015Date of Patent: March 16, 2021Assignees: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HAVARD COLLEGEInventors: Feng Zhang, Le Cong, Fei Ran, Matthias Heidenreich, Lukasz Swiech
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Publication number: 20200283743Abstract: In one aspect, embodiments disclosed herein are directed to engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector proteins a Type II effector protein. In certain other example embodiments, the Type V effector protein is Cas9 or an orthologs or engineered variant thereof. Example Cas9 proteins suitable for use in the embodiments disclosed herein are discussed in further detail below.Type: ApplicationFiled: August 17, 2017Publication date: September 10, 2020Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Feng ZHANG, David Arthur SCOTT, Winston Xia YAN, Sourav CHOUDHURY, Matthias HEIDENREICH
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Publication number: 20200263190Abstract: The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA or RNA-targeting systems comprising a novel DNA or RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.Type: ApplicationFiled: April 19, 2017Publication date: August 20, 2020Inventors: Feng Zhang, Bernd Zetsche, Matthias Heidenreich, Sourav Choudhury
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Publication number: 20200080112Abstract: Embodiments disclosed herein are directed to engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector protein is a Type V effector protein. In certain other example embodiments, the Type V effector protein is Cpf1. Embodiments disclosed herein are directed to viral vectors for delivery of CRISPR-Cas effector proteins, including Cpf1. In certain example embodiments, the vectors are designed so as to allow packaging of the CRISPR-Cas effector protein within a single vector. There is also an increased interest in the design of compact promoters for packing and thus expressing larger transgenes for targeted delivery and tissue-specificity.Type: ApplicationFiled: August 17, 2017Publication date: March 12, 2020Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGYInventors: Feng ZHANG, David Arthur SCOTT, Winston Xia YAN, Sourav CHOUDHURY, Matthias HEIDENREICH
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Publication number: 20190218276Abstract: Transcriptomes of individual neurons provide rich information about cell types and dynamic states. However, it is difficult to capture rare dynamic processes, such as adult neurogenesis, because isolation from dense adult tissue is challenging, and markers for each phase are limited. Here, Applicants developed Nuc-seq, Div-Seq, and Dronc-Seq. Div-seq combines Nuc-Seq, a scalable single nucleus RNA-Seq method, with EdU-mediated labeling of proliferating cells. Nuc-Seq can sensitively identify closely related cell types within the adult hippocampus. Div-Seq can track transcriptional dynamics of newborn neurons in an adult neurogenic region in the hippocampus. Dronc-Seq uses a microfluidic device to co-encapsulate individual nuclei in reverse emulsion aqueous droplets in an oil medium together with one uniquely barcoded mRNA-capture bead. Finally, Applicants found rare adult newborn GABAergic neurons in the spinal cord, a non-canonical neurogenic region.Type: ApplicationFiled: October 27, 2016Publication date: July 18, 2019Applicants: THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTITUTE OF TECHNOLOGY, PRESIDENT AND FELLOWS OF HARVARD COLLEGEInventors: Aviv Regev, Feng Zhang, Naomi Habib, Yinqing Li, Matthias Heidenreich, Lukasz Swiech, Anindita Basu, David Weitz, Inbal Avraham Davidi
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Publication number: 20160340661Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: ApplicationFiled: June 10, 2016Publication date: November 24, 2016Inventors: Le Cong, David Benjamin Turitz Cox, Matthias Heidenreich, Randall Jeffrey Platt, Lukasz Swiech, Feng Zhang
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Publication number: 20160175462Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: ApplicationFiled: December 16, 2015Publication date: June 23, 2016Inventors: Feng ZHANG, Le CONG, Fei RAN, Matthias HEIDENREICH, Lukasz SWIECH
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Publication number: 20160153004Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which include post mitotic cells which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.Type: ApplicationFiled: December 16, 2015Publication date: June 2, 2016Inventors: Feng ZHANG, Matthias HEIDENREICH, Lukasz SWIECH