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Showing 1 to 3 of 3 for “"Spinal and bulbar muscular atrophy"”.

  1. Modelling neurodegenerative diseases in human iPSC-derived neurons

    … pathology shared by a varied class of diseases, and many of the mutations that are known to cause such diseases have been linked to protein aggregation and autophagy dysfunction. Improvements to gene editing and neuronal differentiation strategies have enabled the derivation of in vitro disease …

    cambridge Repository record for Modelling neurodegenerative diseases in human iPSC-derived neurons (opens in a new tab)

  2. Physiological and pathological role of serine 96 phosphorylation in the regulation of androgen receptor

    Spinal and bulbar muscular atrophy (SBMA) is an X-linked neuromuscular disorder characterized by the progressive dysfunction and loss of lower motor neurons. SBMA is caused by the expansion of a CAG tandem repeat encoding a polyglutamine (polyQ) tract in the androgen receptor (AR) gene. SBMA …

    trento Repository record for Physiological and pathological role of serine 96 phosphorylation in the regulation of androgen receptor (opens in a new tab)

  3. AR-A IPSCS: GENERATION OF AN ADVANCED CELLULAR MODEL TO INVESTIGATE A NOVEL THERAPEUTIC APPROACH FOR SBMA

    Spinal and Bulbar Muscular Atrophy (SBMA) is a rare, highly disabling, neurodegenerative disease that unfortunately still lacks an effective treatment to date. Patients affected by SBMA present muscular impairment, characterized by cramps and muscle twitching in the early stages and by difficulty …

    milano Repository record for AR-A IPSCS: GENERATION OF AN ADVANCED CELLULAR MODEL TO INVESTIGATE A NOVEL THERAPEUTIC APPROACH FOR SBMA (opens in a new tab)