Novel diagnostic and treatment method for Alzheimer's disease
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Jerry Kokoshka
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Therapeutic for other neurodegenerative disorders, including Welander distal myopathy (WDM), a rare autosomal dominant disorder caused by a missense mutation in the human TIA1 gene. Small molecules penetrate the CNS and pass through cell membranes (unlike main competitors, which are antibody-based). Most current anti-tau therapies in development are immunotherapies, which have thus far been ineffective, in large part because of poor antibody penetration into the CNS and poor membrane permeability.
Miranda AM, Ashok A, Chan RB, Zhou B, Xu Y, McIntire LB, Area-Gomez E, Di Paolo G, Duff KE, Oliveira TG, Nuriel T.
Presenilin mutations linked to familial Alzheimer’s disease cause an imbalance in phosphatidylinositol 4,5-bisphosphate metabolism Proc Natl Acad Sci USA.
- 6Neurodegeneration
- 3Tau protein
- 3Amyloid beta
- 3Blood–brain barrier
- 3Small molecule
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- 8Biology
- 8Branches of biology
- 6Neuroscience
- 5Cell biology
- 4Medicine
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- 3Donald William Landry MD, Ph.D.
- 3Laura Beth Johnson McIntire
- 3Shi-xian Deng
- 3Tae-Wan Kim Ph.D.
- 2Joseph B. Rayman Ph.D.
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- 8Jerry Kokoshka
- 7Kristin Neuman
- 6Sara Gusik
- 2Joan Martinez
- 1Dovina Qu
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- 5Pathology
- 3Medicine
- 2Neuroscience
- 1Biochemistry & Molecular Biophysics
- 1Biomedical Engineering
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- 7Columbia University Medical Center (CUMC)
- 2College of Physicians and Surgeons (CUMC
- 1Fu Foundation School of Engineering and Applied Science (SEAS)
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