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X-ALD Patients Hope to See Results With Cell-Based Gene Therapy

Mar 31, 2020 10:06:00 AM / by Stacy Matthews Branch, DVM, PhD posted in Gene Therapy, Bone Marrow, Cell Therapy, CNS/Neurodegeneration

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As Phase 3 clinical trials begin for Lenti-D as a treatment for patients with X-ALD, the drug also shows promise for treating a range of neurodegenerative diseases.

X‐linked adrenoleukodystrophy (X‐ALD) is a peroxisomal disorder resulting from a mutation in the human ATP-binding cassette, sub-family D, member 1 gene (ABCD1). The ABCD1 gene codes for the adrenoleukodystrophy protein (ALDP) needed for very-long-chain fatty acids (VLCFAs) to enter peroxisomes for degradation. When this process is disrupted, the VLCFAs accumulate in various tissues of the body, including the brain. VLCFA accumulation and demyelination of brain white matter lead to the severe neurological symptoms seen with X-ALD.

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How Transplanted Human Bone Marrow Cells May Improve ALS Therapy

Jul 9, 2019 10:07:00 AM / by Stacy Matthews Branch, DVM, PhD posted in Bone Marrow, CNS/Neurodegeneration

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While no cure exists for ALS, researchers are finding ways to use unmodified human bone marrow CD34+ cells for spinal restoration.

The renowned physicist Stephen Hawking influenced how countless scientists worldwide view the universe. He also demonstrated his remarkable ability to survive amyotrophic lateral sclerosis (ALS) for 55 years. He is thought to be the longest survivor of ALS. According to the ALS Association, 50% of those with ALS survive for about 3 years after diagnosis, 20% survive for 5 or more years, and 10% survive for 10 years or more. There is no cure for ALS, and therapies that can slow or delay progression, decrease symptoms, and repair damaged spinal-cord tissue are continuously sought.

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Young Blood's Effect on Alzheimer’s Disease

Jan 22, 2018 10:23:00 AM / by Stacy Matthews Branch, DVM, PhD posted in CNS/Neurodegeneration, Plasma

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In Alzheimer's disease, brain cells die, and their connections to one another degenerate leading to decline in mental function. The brain changes that occur in Alzheimer's disease are associated with the production and deposition of beta-amyloid plaques. Currently, treatment of Alzheimer's disease focuses on delaying or slowing mental decline and addressing behavioral complications. There are a few U.S. FDA−approved drugs used to treat Alzheimer's disease symptoms. These drugs regulate neurotransmitters, but have limited duration, only work in some patients, and do not address the underlying cause.

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p11 Levels Increase in Peripheral Blood Mononuclear Cells from Patients with Parkinson’s Disease

Nov 9, 2016 12:00:00 AM / by Stacy Matthews Branch, DVM, PhD posted in p11, PBMCs, stem cell research, Stem Cell Therapy, CNS/Neurodegeneration, Basic Research

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Depression is a common and challenging component of Parkinson’s disease (PD). Up to 70% of patients with PD experience depression. This clinical symptom is not only a result of PD, but a part of the condition itself. Study findings have shown that inflammation not only plays a part in the physical signs of PD, but is also involved in the development of depression. The increase in inflammatory mediators, such as cytokines, in PD is detectable before signs of PD begin. These mediators may serve as important biomarkers of PD that can be utilized to detect PD and allow early intervention.

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Maternal T cells Gone Rogue: A New Link Between Immune Activation And Autism

Apr 13, 2016 1:00:11 PM / by Dalia Gaddis posted in T Cells, CNS/Neurodegeneration, Basic Research

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Scientists find that IL-17 produced by maternal T cells may cause brain damage to their infants and lead to autism.

Are a mother’s own T cells, which are designed to protect her and her unborn infant, to blame for autism? There might be an association between excessive maternal T cells activation and autism.

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