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    Muscular Dystrophy

    Muscular Dystrophy is caused when atypical gene mutations impede the production of proteins essential to the formation of healthy muscle. The main symptoms of the group of diseases designated as muscular dystrophy are loss of muscle mass and weakness. Medication can be prescribed to reduce symptoms but there is no known cure.

    Webinars

    • APR 11, 2023 | 8:00 AM
      Webinar: Dystrophin Quantification in Preclinical and Clinical Settings
      Webinar: Dystrophin Quantification in Preclinical and Clinical...
      Date: April 11, 2023 Time: 8:00am (PDT), 11:00am (EDT), 5:00pm (CEST) Duchenne muscular dystrophy is caused by mutations in the dystrophin encoding DMD gene that disrupt the reading frame. M...
      Speaker: Annemieke Aartsma-Rus, PhD
      Sponsored By: Bio-Techne
      18
      SEP 29, 2021 | 11:59 PM
      Nanoparticle Based Delivery of Gene Editing Enzymes
      Nanoparticle Based Delivery of Gene Editing Enzymes
      Cas9 based therapeutics have the potential to revolutionize the treatment of genetic diseases. However, safe and effective methods for delivering Cas9 protein, gRNA and donor DNA need to be...
      Speaker: Niren Murthy, PhD
      Presented at: CRISPR Virtual Event Series 2021
      2 2
      SEP 30, 2020 | 12:00 AM
      In vivo delivery of Cas9 ribonucleoprotein and donor DNA with gold nanoparticles
      In vivo delivery of Cas9 ribonucleoprotein and donor DNA with...
      Cas9 based therapeutics have the potential to revolutionize the treatment of genetic diseases. However, safe and effective methods for delivering Cas9 protein, gRNA and donor DNA need to be...
      Speaker: Niren Murthy
      Presented at: CRISPR Virtual Event Series 2020
      4
      JUL 02, 2020 | 1:30 PM
      Molecular Diagnosis of Genetic Diseases using CE in an era of different technologies
      Molecular Diagnosis of Genetic Diseases using CE in an era of...
      Genetic diseases are known occur due to various types of mutations in the human genome. The molecular assays to diagnose these diseases have been developed based on the type of mutation comm...
      Speaker: Ashwin Dalal, MD
      Presented at: APJ Virtual Genetic Solutions Tour 2020
      3
      NOV 14, 2019 | 6:00 AM
      High Resolution View of D4Z4 Repeat Regions for Studying Facioscapulohumeral Muscular Dystrophy (FSHD) Using Whole Genome Optical Mapping
      High Resolution View of D4Z4 Repeat Regions for Studying Facioscapulohumeral...
      Facioscapulohumeral muscular dystrophy (FSHD), one of the most common forms of progressive muscular dystrophies and is often conventionally diagnosed by Southern blot analysis. The accurate d...
      Speaker: Alka Chaubey, PhD, FACMG
      Presented at: Clinical Diagnostics & Research Virtual Event Series 2019
      Sponsored By: Bionano
      2
      JUL 01, 2017 | 12:00 AM
      Pluripotent stem cells to explore mechanisms and treatments of neuromuscular diseases
      Pluripotent stem cells to explore mechanisms and treatments of...
      Disease-specific human pluripotent stem cells (hPSCs) represent a new chance to unravel cellular and molecular mechanisms of neurological diseases. Along this line, we were among the first to...
      Speaker: Cécile Martinat
      Presented at: 5th annual 24 Hours of Stem Cellsâ„¢ virtual event
      2
      MAR 16, 2017 | 1:30 PM
      Humans and fish to study the cellular mechanisms of neuromuscular development
      Humans and fish to study the cellular mechanisms of neuromuscular...
      Studying the genetics of rare congenital disorders disrupting cognitive function has led to the identification of multiple disease genes that helped us better understand the mechanisms underl...
      Speaker: Chiara Manzini, PhD
      Presented at: Neuroscience Virtual Event Series 2017
      1
      AUG 30, 2016 | 8:00 AM
      Disease modeling in pluripotent stem cell-derived cardiomyocytes
      Disease modeling in pluripotent stem cell-derived cardiomyocytes
      Over the last 15 years, human pluripotent stem cell (hPSC) technologies have progressed from academic curiosities into tools with the promise to underpin commerce, leading to real progress in...
      Speaker: Chris Denning, PhD
      Presented at: 4th Annual 24 Hours of Stem Cellsâ„¢ virtual event
      2
      SEP 24, 2014 | 6:00 AM
      Translational Medicine for Rare Disease: Lessons from Duchenne Muscular Dystrophy
      Translational Medicine for Rare Disease: Lessons from Duchenne...
      Duchenne Muscular Dystrophy (DMD) is an X-linked genetic disease occurring in 1 in 3,500 births, and is uniformly fatal in boys. It is caused by any one of over 50 known mutations in the gen...
      Speaker: Susan Ward, PhD
      Presented at: Advances in Drug Discovery and Development
      14
    APR 11, 2023 | 8:00 AM
    Webinar: Dystrophin Quantification in Preclinical and Clinical Settings
    Webinar: Dystrophin Quantification in Preclinical and Clinical...
    Date: April 11, 2023 Time: 8:00am (PDT), 11:00am (EDT), 5:00pm (CEST) Duchenne muscular dystrophy is caused by mutations in the dystrophin encoding DMD gene that disrupt the reading frame. M...
    Speaker: Annemieke Aartsma-Rus, PhD
    Sponsored By: Bio-Techne
    18
    SEP 29, 2021 | 11:59 PM
    Nanoparticle Based Delivery of Gene Editing Enzymes
    Nanoparticle Based Delivery of Gene Editing Enzymes
    Cas9 based therapeutics have the potential to revolutionize the treatment of genetic diseases. However, safe and effective methods for delivering Cas9 protein, gRNA and donor DNA need to be...
    Speaker: Niren Murthy, PhD
    Presented at: CRISPR Virtual Event Series 2021
    2 2
    SEP 30, 2020 | 12:00 AM
    In vivo delivery of Cas9 ribonucleoprotein and donor DNA with gold nanoparticles
    In vivo delivery of Cas9 ribonucleoprotein and donor DNA with...
    Cas9 based therapeutics have the potential to revolutionize the treatment of genetic diseases. However, safe and effective methods for delivering Cas9 protein, gRNA and donor DNA need to be...
    Speaker: Niren Murthy
    Presented at: CRISPR Virtual Event Series 2020
    4
    JUL 02, 2020 | 1:30 PM
    Molecular Diagnosis of Genetic Diseases using CE in an era of different technologies
    Molecular Diagnosis of Genetic Diseases using CE in an era of...
    Genetic diseases are known occur due to various types of mutations in the human genome. The molecular assays to diagnose these diseases have been developed based on the type of mutation comm...
    Speaker: Ashwin Dalal, MD
    Presented at: APJ Virtual Genetic Solutions Tour 2020
    3
    NOV 14, 2019 | 6:00 AM
    High Resolution View of D4Z4 Repeat Regions for Studying Facioscapulohumeral Muscular Dystrophy (FSHD) Using Whole Genome Optical Mapping
    High Resolution View of D4Z4 Repeat Regions for Studying Facioscapulohumeral...
    Facioscapulohumeral muscular dystrophy (FSHD), one of the most common forms of progressive muscular dystrophies and is often conventionally diagnosed by Southern blot analysis. The accurate d...
    Speaker: Alka Chaubey, PhD, FACMG
    Presented at: Clinical Diagnostics & Research Virtual Event Series 2019
    Sponsored By: Bionano
    2
     
     
  • JUL 01, 2017 | 12:00 AM
    Pluripotent stem cells to explore mechanisms and treatments of neuromuscular diseases
    Pluripotent stem cells to explore mechanisms and treatments of...
    Disease-specific human pluripotent stem cells (hPSCs) represent a new chance to unravel cellular and molecular mechanisms of neurological diseases. Along this line, we were among the first to...
    Speaker: Cécile Martinat
    Presented at: 5th annual 24 Hours of Stem Cellsâ„¢ virtual event
    2
    MAR 16, 2017 | 1:30 PM
    Humans and fish to study the cellular mechanisms of neuromuscular development
    Humans and fish to study the cellular mechanisms of neuromuscular...
    Studying the genetics of rare congenital disorders disrupting cognitive function has led to the identification of multiple disease genes that helped us better understand the mechanisms underl...
    Speaker: Chiara Manzini, PhD
    Presented at: Neuroscience Virtual Event Series 2017
    1
    AUG 30, 2016 | 8:00 AM
    Disease modeling in pluripotent stem cell-derived cardiomyocytes
    Disease modeling in pluripotent stem cell-derived cardiomyocytes
    Over the last 15 years, human pluripotent stem cell (hPSC) technologies have progressed from academic curiosities into tools with the promise to underpin commerce, leading to real progress in...
    Speaker: Chris Denning, PhD
    Presented at: 4th Annual 24 Hours of Stem Cellsâ„¢ virtual event
    2
    SEP 24, 2014 | 6:00 AM
    Translational Medicine for Rare Disease: Lessons from Duchenne Muscular Dystrophy
    Translational Medicine for Rare Disease: Lessons from Duchenne...
    Duchenne Muscular Dystrophy (DMD) is an X-linked genetic disease occurring in 1 in 3,500 births, and is uniformly fatal in boys. It is caused by any one of over 50 known mutations in the gen...
    Speaker: Susan Ward, PhD
    Presented at: Advances in Drug Discovery and Development
    14
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