Rare disease research news from UMass Medical School
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Jun 7, 2022Read more
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Apr 21, 2022
UMass Chan research supports development of new suppressor-tRNA-based gene therapies
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Mar 17, 2022
Muscle stem cell technology developed at UMass Chan prelude to new muscular dystrophy therapeutics
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Feb 14, 2022
First gene therapy for Tay-Sachs disease successfully given to two children
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Feb 10, 2022
Raiden Science Foundation makes gift to UMass Chan for UBA5 gene therapy research
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Feb 10, 2022
UMass Chan launches Translational Institute for Molecular Therapeutics
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Dec 23, 2021
UMass Chan clinical trial shows antisense oligonucleotide safely suppresses mutant ALS gene in pilot human study
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Nov 18, 2021
Flotte lab to develop gene therapy models for genetic lung disease
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Nov 2, 2021
Sio Gene Therapies reports positive interim data for gene therapy trial
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Oct 4, 2021
Riaan Research Initiative funds Cockayne syndrome gene replacement therapy research at UMass Chan Medical School
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Aug 26, 2021
Behind the research: Jae-Hyuck Shim strives to unlock cure for devastating childhood bone disease
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Aug 17, 2021
Two new studies from Daryl Bosco lab shed new light on ALS mechanisms
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Jul 29, 2021
Guangping Gao and Dan Wang partner with ASC Therapeutics to develop novel gene therapy for maple syrup urine disease
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Apr 27, 2021
USA Today reports on first baby in gene therapy clinical trial for Sandhoff, Tay-Sachs diseases
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Apr 21, 2021
FDA clears investigational new drug application for ALS gene therapy candidate
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Apr 8, 2021
UMMS establishes gene therapy collaborative research agreement with Pfizer
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Apr 7, 2021
Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium
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Mar 3, 2021
Innovative neurosurgical technique used in Tay-Sachs gene therapy clinical trial
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Feb 3, 2021
First patient dosed in clinical trial of gene therapy for Tay-Sachs and Sandhoff diseases
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Nov 9, 2020
FDA clears IND application for Tay-Sachs gene therapy
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