Results 11 to 20 of about 1,504,225 (383)
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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Lifting the differentiation embargo [PDF]
, 2016 Effective differentiation therapy for acute myeloid leukemia (AML) has been restricted to a small subset of patients with one defined genetic abnormality. Using an unbiased small molecule screen, Sykes et al.
Holyoake, Tessa, Latif, Anne-Louise
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DNA Edition: ad portas of a revolution in genetic manipulation
Iatreia, 2020 In biological sciences, genetic therapy constitutes a “trend topic” since its beginning. Development of new technologies in bioengineering as zinc-finger nucleases (ZFN), Transcription activator-like effector nu-cleases (TALEN) and ...
Madariaga Perpiñán, Ithzayana +3 more
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The use of genes for performance enhancement: doping or therapy? [PDF]
, 2011 Recent biotechnological advances have permitted the manipulation of genetic sequences to treat several diseases in a process called gene therapy. However, the advance of gene therapy has opened the door to the possibility of using genetic manipulation ...
Collares, T. F. +4 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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