 |
|
Gene therapy slows progression of fatal neurodegenerative disease in children
May 14, 2008Gene therapy to replace the faulty CLN2 gene, which causes a neurodegenerative disease that is fatal by age 8-12 years, was able to slow significantly the rate of neurologic decline in treated children, according to a paper published online ahead of print in the May 2008 issue (Vol. 19 No. 5) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The paper is available free online at www.liebertpub.com/hum
Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) is an autosomal recessive genetic disorder that causes degeneration of the central nervous system. It is a form of Batten disease, a group of lysosomal storage disease in which a lipofuscin-like material is not broken down and accumulates in neurons, causing cognitive impairment, visual failure, seizures, and progressive deterioration of motor function.
Ronald Crystal and colleagues from Weill Cornell Medical College (New York, NY), describe a study conducted in 10 children with LINCL who received gene therapy to replace the defective CLN2 gene via administration of human CLN2 carried in an adeno-associated virus (AAV). In the paper entitled "Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis with CNS Administration of a Serotype 2 Adeno-associated virus expressing the CLN2 cDNA," the authors report that over an 18-month period, assessment using a neurologic rating scale demonstrated significant slowing of disease progression in the treated, compared to the untreated children. On the basis of these findings, the authors proposed that additional studies to assess the safety and efficacy of AAV-mediated gene therapy for LINCL be pursued.
Although the treatment was associated with some serious adverse events in some patients, these were not unequivocally attributable to the gene therapy vector.
"This clinical trial is an important step toward the development of treatments for this group of underserved inherited neurodegenerative disorders," says James M. Wilson, MD, PhD, Editor-in-Chief and Head of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, at the University of Pennsylvania School of Medicine, in Philadelphia.
Mary Ann Liebert, Inc./Genetic Engineering News
Although the treatment was associated with some serious adverse events in some patients, these were not unequivocally attributable to the gene therapy vector.
"This clinical trial is an important step toward the development of treatments for this group of underserved inherited neurodegenerative disorders," says James M. Wilson, MD, PhD, Editor-in-Chief and Head of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, at the University of Pennsylvania School of Medicine, in Philadelphia.
Mary Ann Liebert, Inc./Genetic Engineering News
Neurodegenerative Disease Current Events and Neurodegenerative Disease News RSSNovel mouse gene reduces major pathologies associated with Alzheimer's disease
A new study reveals that a previously undiscovered mouse gene reduces the two major pathological perturbations commonly associated with Alzheimer's disease (AD).
Mouse gene suppresses Alzheimer's plaques and tangles
Investigators at Burnham Institute for Medical Research (Burnham) and colleagues have identified a novel mouse gene (Rps23r1) that reduces the accumulation of two toxic proteins that are major players in Alzheimer's disease: amyloid beta and tau.
Widely used cholesterol-lowering drug may prevent progression
Simvastatin, a commonly used, cholesterol-lowering drug, may prevent Parkinson's disease from progressing further. Neurological researchers at Rush University Medical Center conducted a study examining the use of the FDA-approved medication in mice with Parkinson's disease and found that the drug successfully reverses the biochemical, cellular and anatomical changes caused by the disease.
Member of NFL Hall of Fame diagnosed with degenerative brain disease
The Center for the Study of Traumatic Encephalopathy (CSTE) at Boston University School of Medicine (BUSM) announced today that a recently deceased member of the NFL Hall of Fame suffered from the degenerative brain disease Chronic Traumatic Encephalopathy (CTE) when he died, becoming the 10th former NFL player diagnosed with the disease.
First former college football player diagnosed with CTE
The Center for the Study of Traumatic Encephalopathy (CSTE) at Boston University School of Medicine (BUSM) announced today that a deceased former college football player who died at age 42 was already suffering from the degenerative brain disease, Chronic Traumatic Encephalopathy (CTE).
Natural compounds, chemotherapeutic drugs may become partners in cancer therapy
Research in the Linus Pauling Institute at Oregon State University suggests that some natural food compounds, which previously have been studied for their ability to prevent cancer, may be able to play a more significant role in treating it - working side-by-side with the conventional drugs that are now used in chemotherapy.
August 10, 2009 New Class of Compounds Discovered for Potential Alzheimer's Disease Drug, Penn Study Finds
A new class of molecules capable of blocking the formation of specific protein clumps that are believed to contribute to the dementia of Alzheimer's disease (AD) patients has been discovered by researchers at the University of Pennsylvania School of Medicine.
Caffeine reverses memory impairment in Alzheimer's mice
Coffee drinkers may have another reason to pour that extra cup. When aged mice bred to develop symptoms of Alzheimer's disease were given caffeine - the equivalent of five cups of coffee a day - their memory impairment was reversed.
Huntington's disease deciphered
Researchers at the University of Illinois at Chicago College of Medicine have discovered how the mutated huntingtin gene acts on the nervous system to create the devastation of Huntington's disease.
What separates dangerous blood vessel plaques from benign ones
Researchers say they have evidence to explain what separates your average blood vessel plaque from those that are at high risk for triggering the development of dangerous-even fatal-blood clots.
More Neurodegenerative Disease Current Events and Neurodegenerative Disease News Articles
 |
Neurodegenerative Diseases: Neurobiology, Pathogenesis and Therapeutics by M. Flint Beal (Author), Anthony E. Lang (Author), Albert C. Ludolph (Author) Neurodegenerative diseases are major contributors to disability and disease, with Alzheimer's and Parkinson's diseases the most prevalent. This major reference reviews the rapidly advancing knowledge of pathogenesis and treatment of neurodegenerative diseases in the context of a comprehensive survey of each disease and its clinical features. The editors and contributors are among the leading experts in the field internationally. Covering basic science, diagnostic tools and therapeutic approaches, the book focuses on all aspects of neurodegenerative disease, including the normal aging process. The dementias, prion diseases, Parkinson's disease and atypical parkinsonisms, neurodegenerative ataxias, motor neuron diseases, degenerative diseases with chorea, iron and copper disorders, and... |
 |
Neurodegenerative Diseases and Metal Ions: Metal Ions in Life Sciences by Astrid Sigel (Editor), Helmut Sigel (Editor), Roland K. O. Sigel (Editor) About the Series... Metal Ions in Life Sciences links coordination chemistry and biochemistry in their widest sense and thus increases our understanding of the relationship between the chemistry of metals and life processes. The series reflects the interdisciplinary nature of Biological Inorganic Chemistry and coordinates the efforts of scientists in fields like biochemistry, inorganic chemistry, coordination chemistry, molecular and structural biology, enzymology, environmental chemistry, physiology, toxicology, biophysics, pharmacy, and medicine. Consequently, the volumes are an essential source for researchers active in these and related fields as well as teachers preparing courses, e.g., in Bioinorganic Chemistry. About this Book... Volume 1, devoted... |
 |
Oxidative Stress in Cancer, AIDS, and Neurodegenerative Diseases (Oxidative Stress and Disease, 1) by Montagnier (Author) Presents basic, chemical, biological, and medical studies of free radicals on different targets and the consequences of their reactivity-covering the chemistry and biochemistry of free radicals, free radicals as second messengers that group the activation of transcription factors and enzymes, the importance of the antioxidant system in cell metabolism regulation, and the role of free radicals and antioxidants in disease management. Written by over 200 international authorities, the book offers incisive discussions on the significance of oxidant-antioxidant balance and cytotoxicity of free radicals when produced in excess by several sources, specifically radiation hydrogen peroxide as a stimulant of transcription factor NFkB leading to cell response against oxidative stress ... |
 |
Fatal Attractions: Protein Aggregates in Neurodegenerative Disorders (Research and Perspectives in Alzheimer's Disease) by V.M.-Y. Lee (Editor), J.Q. Trojanowski (Editor), L. Buee (Editor), Y. Christen (Editor) In this volume are contributions based on a meeting arranged by the WHO and the Fondation IPSEN. The scientists focus on neurodegenerative disorders like Alzheimer's Disease, Chromosome 17-Linked Dementia, Parkinson's Disease and disorders with tauopathies. |
 |
So Much So Fast Starring: Stephen Heywood, Jamie Heywood Directed By: Steven Ascher;Jeanne Jordan What would you do if you were 29 and found you may only have a few years to live? So Much So Fast is about the remarkable events set in motion when Stephen Heywood discovers he has ALS (Lou Gehrig's disease) and his brother Jamie becomes obsessed with finding a cure. |
 |
Glutamine Repeats and Neurodegenerative Diseases: Molecular Aspects by Peter S. Harper (Editor), Max Perutz (Editor) (The Royal Society) Univ. of Wales, Cardiff, UK. Molecular biology text focuses on the discovery of a common pathogenic basis for a group of genetically inherited disorders, including Huntington's Disease. Discusses how these diseases share certain molecular similarities, what causes neurodegeneration to begin, and other important discoveries. For researchers and clinicians. |
 |
Molecular Mechanisms of Neurodegenerative Diseases (Contemporary Clinical Neuroscience) by Marie-Francoise Chesselet (Editor) Univ. of California, Los Angeles. First volume in this series. Synthesizes novel ideas and concepts emerging to create a fresh understanding of neurodegenerative disorders, details mechanistic similarities in Alzheimer's, Parkinson's, and related diseases. DNLM: Neurodegenerative Diseases--genetics. |
 |
Heat Shock Proteins and the Brain: Implications for Neurodegenerative Diseases and Neuroprotection (Heat Shock Proteins) by Alexzander A.A. Asea (Author), Alexzander A.A. Asea (Editor), Ian R. Brown (Editor) With the prevalence of neurodegenerative diseases on the rise as average life expectancy increases, the hunt for effective treatments and preventive measures for these disorders is a pressing challenge. Neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease and amyotrophic lateral sclerosis have been termed 'protein misfolding disorders' that are characterized by the neural accumulation of protein aggregates. Manipulation of the cellular stress response involving the induction of heat shock proteins offers a therapeutic strategy to counter conformational changes in neural proteins that trigger pathogenic cascades resulting in neurodegenerative diseases. Heat shock proteins are protein repair agents that provide a line of defense against misfolded,... |
 |
Intracellular Traffic and Neurodegenerative Disorders (Research and Perspectives in Alzheimer's Disease) by Peter H. St.George-Hyslop (Editor), William C. Mobley (Editor), Yves Christen (Editor) This book explores the role of sub-cellular trafficking in the pathogenesis, treatment and prevention of neurodegenerative diseases. Recent findings point to faulty trafficking as contributing to the dysfunction and degeneration of neurons and neural circuits. Increasingly, research is targeting the mechanisms responsible and means to address them therapeutically. This book investigates the ways in which sub-cellular trafficking pathways play a role both in the pathological accumulation of these misfolded proteins and in attempts to clear them. Because subcellular protein trafficking has an important role in the biology of neuronal function and survival, this also addresses how trophic factors maintain cell, including cell interactions and how the underlying mechanisms may be compromised... |
|
ADULTS WITH NEURODEGENERATIVE DISEASES: Occupational Therapy Practice Guidelines (Aota Practice Guidelines) by Susan Forwell (Author) |
| © 2009 BrightSurf.com |