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    On Demand Webinars

    AAV Based Gene Therapy for Rare Genetic Disorders: Strategies and Challenges

    Variable penetrance has long posed a challenge for predicting the severity of a disease
    Genetic Disorders

    Genetic Diseases More Severe with Adverse Coding, Regulatory Variant Combos

    Genetic Disorders

    Study Finds Healthy Respiratory Disease Gene Carriers Have Greater Breathing Capacity

    Muscle Stem Cells
    Genetic Disorders

    CRISPR’d Stem Cells Could Provide Enduring Therapy for Muscular Dystrophy

    Genetic Disorders

    Rare Genetic Variants from Birth Have Negative Effect on Lifespan

    A view of the eye of a dog affected by the canine equivalent of Best disease before (left) and five years after (right) the gene therapy underscores how the treatment reversed a sizeable lesion—and lasted. The black mark visible on the treated eye is the injection site. [University of Pennsylvania]
    Genetic Disorders

    Gene-Therapy Trials for Inherited Form of Blindness Could Start in Two...

    Genetic Disorders

    Scholar Rock: A New Twist on Targeting Growth Factors

    Genetic Disorders

    Peptide Nucleic Acids Used to Edit Genes, Cure Mice of Inherited...

    Genetic Disorders

    Link Explained between Down Syndrome and Leukemia

    Genetic Disorders

    Sartorius and McMaster Partner to Advance Next-Gen Biomanufacturing

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