Results 1 to 10 of about 1,623,366 (311)
Biomimetic Peptides: A New Generation of Gene Transfer Vectors [PDF]
مجله دانشکده پزشکی اصفهان, 2022 Gene therapy is a new approach that aims to modify defective genes or intracellular expression of therapeutic proteins, and this depends on the use of high-efficiency gene transfer systems.
Hooman Mahmoudi Aznaveh, Maryam Nikkhah
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Molecular Therapy: Methods & Clinical Development, 2021 Adeno-associated virus (AAV) vectors are considered efficient vectors for gene transfer, as illustrated by recent successful clinical trials targeting retinal or neurodegenerative disorders.
Gwladys Gernoux +12 more
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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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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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مجلة بغداد للعلوم, 2020 Background: Adipose derived-mesenchymal stem cells have been used as an alternative to bone marrow cells in this study. Objective: We investigated the in vitro isolation, identification, and differentiation of stem cells into neuron cells, in order to ...
Ahmed Majeed Al-Shammari +4 more
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Human Neural Stem Cells: Translational Research for Neonatal Hypoxic-Ischemic Brain Injury [PDF]
Neonatal Medicine, 2019 Neonatal hypoxic-ischemic (HI) brain injury is a major cause of neonatal mortality and long-term neurodevelopmental disabilities. Although promising neuroprotective interventions have been studied, the current management of HI brain injury has been ...
Jeong Eun Shin +4 more
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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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A review of Genetic Etiology and Emerging Molecular Therapies for FSHD in Preclinical Studies [PDF]
Reviews in Clinical Medicine, 2021 Facioscapulohumeral muscular dystrophy is one of the most common musculoskeletal diseases with a considerable burden. Most of the affected individuals experience muscle weakness as the common muscular symptom.
Mohammad Reza Seyyed taghia +4 more
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Frontiers in Medicine, 2023 ObjectiveOsteoarthritis (OA) is the most common degenerative joint disease, characterized by a progressive loss of cartilage associated with synovitis and subchondral bone remodeling.
Melanie Uebelhoer +7 more
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Genetic therapies against HIV [PDF]
Nature Biotechnology, 2007 Highly active antiretroviral therapy prolongs the life of HIV-infected individuals, but it requires lifelong treatment and results in cumulative toxicities and viral-escape mutants. Gene therapy offers the promise of preventing progressive HIV infection by sustained interference with viral replication in the absence of chronic chemotherapy.
John J, Rossi +2 more
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