Results 11 to 20 of about 835,919 (314)
Genetic therapies for neurological disorders [PDF]
Human Genetics, 2021 AbstractIn recent years, it has become increasingly apparent that many neurological disorders are underpinned by a genetic aetiology. This has resulted in considerable efforts to develop therapeutic strategies which can treat the disease-causing mutation, either by supplying a functional copy of the mutated gene or editing the genomic sequence. In this
Laura FitzPatrick, Adrian Bird
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Achromatopsia: Genetics and Gene Therapy [PDF]
Molecular Diagnosis & Therapy, 2021 Achromatopsia (ACHM), also known as rod monochromatism or total color blindness, is an autosomal recessively inherited retinal disorder that affects the cones of the retina, the type of photoreceptors responsible for high-acuity daylight vision. ACHM is caused by pathogenic variants in one of six cone photoreceptor-expressed genes.
Stylianos Michalakis +4 more
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Genetic therapy for pain management
Current Review of Pain, 2001 It has been suggested that there are two main approaches to the use of gene therapy in the treatment of chronic disease, which has been confirmed by recent studies in animals.
Steven P. Wilson, David C. Yeomans
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Annual Review of Pathology: Mechanisms of Disease, 2021 Genetic diseases cause numerous complex and intractable pathologies. DNA sequences encoding each human's complexity and many disease risks are contained in the mitochondrial genome, nuclear genome, and microbial metagenome. Diagnosis of these diseases has unified around applications of next-generation DNA sequencing.
Roth, Theodore L, Marson, Alexander
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Genetics and Therapies for GM2 Gangliosidosis [PDF]
Current Gene Therapy, 2018 Tay-Sachs disease, caused by impaired β-N-acetylhexosaminidase activity, was the first GM2 gangliosidosis to be studied and one of the most severe and earliest lysosomal diseases to be described. The condition, associated with the pathological build-up of GM2 ganglioside, has acquired almost iconic status and serves as a paradigm in the study of ...
Cachon-Gonzalez, Maria Begona +2 more
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Cellular and genetic therapies for haemophilia [PDF]
Haemophilia, 2006 Summary. Haemophilia continues to be a prime target for a variety of gene and cell‐based therapies. Pre‐clinical successes in both mouse and dog models of the disease have been documented with a variety of approaches over the past decade, and there have now been six small clinical trials of gene transfer in haemophilia.
Lillicrap, David +2 more
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Biomedicines, 2016 Background: Chemotherapy is one of the antitumor therapies used worldwide in spite of its serious side effects and unsatisfactory results. Many attempts have been made to increase its activity and reduce its toxicity.
Ahmed M. Al-Shammari +8 more
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Biomedicines, 2023 Background: Pathological changes associated with spinal cord injury (SCI) can be observed distant, rostral, or caudal to the epicenter of injury. These remote areas represent important therapeutic targets for post-traumatic spinal cord repair.
Ravil Garifulin +9 more
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Impact of novel hemophilia therapies around the world
Research and Practice in Thrombosis and Haemostasis, 2022 Hemophilia A and B are hereditary bleeding disorders, characterized by factor VIII or IX deficiencies, respectively. For many decades, prophylaxis with coagulation factor concentrates (replacement therapy) was the standard‐of‐care approach in hemophilia.
Margareth C. Ozelo +1 more
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Targeted genetic repair: an emerging approach to genetic therapy [PDF]
Journal of Clinical Investigation, 2003 The last two decades have heralded a remarkable increase in our understanding of the genetic basis of disease and the endogenous mechanisms responsible for the repair of genomic DNA and the processing of RNA. Targeted gene repair is a powerful yet controversial technique developed to direct base changes in chromosomal genes, while RNA repair is an ...
Bruce A. Sullenger
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