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Showing 1 to 3 of 3 for “"gene and cell therapy"”.

  1. Engineered synthetic translational control for next generation mRNA gene therapies

    … mRNA is an emerging therapeutic modality for gene and cell therapy. Unlike their synthetic DNA counterparts, synthetic mRNA has an increased safety profile due to its transient gene expression and ability to express outside of the nucleus. Furthermore, it can be more easily delivered to cells …

    mit Repository record for Engineered synthetic translational control for next generation mRNA gene therapies (opens in a new tab)

  2. Leveraging Genomic Information for Safer Gene Therapy

    <p>Gene therapy holds enormous potential for the treatment of genetic disorders, which are caused by mutations in DNA disrupting normal gene function. These mutations can be monogenic, affecting a single gene, or polygenic, involving multiple genes. Since, the first gene therapy treatment for SCID …

    tenn-hsc Repository record for Leveraging Genomic Information for Safer Gene Therapy (opens in a new tab)

  3. Chemogenetics, Induced Neurons and Pluripotent Stem Cells: Towards Advanced Gene and Cell Therapies Targeting Epilepsy

    The complexity of the central nervous system and existence of the blood-brain barrier often causes difficulties for traditional pharmacological treatments of neurological diseases. This thesis explores the feasibility and potential for novel gene and cell therapy approaches, which hold better …

    lund Repository record for Chemogenetics, Induced Neurons and Pluripotent Stem Cells: Towards Advanced Gene and Cell Therapies Targeting Epilepsy (opens in a new tab)