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Nanoplatform‐Enabled Genetic Interventions for Central Nervous System Disorders: Advances in Delivery Strategies and Therapeutic Potential [PDF]
Central nervous system (CNS) disorders are driven by complex genetic and epigenetic factors. While gene‐based interventions (siRNA, mRNA, CRISPR systems, etc.) hold transformative potential, their clinical application is severely constrained by ...
Fuming Liang+4 more
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Biomimetic Peptides: A New Generation of Gene Transfer Vectors [PDF]
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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A review of Genetic Etiology and Emerging Molecular Therapies for FSHD in Preclinical Studies [PDF]
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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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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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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Human Neural Stem Cells: Translational Research for Neonatal Hypoxic-Ischemic Brain Injury [PDF]
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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RPE65, an abundant membrane-associated protein present in the retinal pigment epithelium (RPE), is a vital retinoid isomerase necessary for regenerating 11-cis-retinaldehyde from all-trans retinol in the visual cycle.
Mirjana Bjeloš+4 more
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DNA Edition: ad portas of a revolution in genetic manipulation
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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Translating the combination of gene therapy and tissue engineering for treating recessive dystrophic epidermolysis bullosa [PDF]
The combination of gene therapy and tissue engineering is one of the most promising strategies for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
A Dakiw Piaceski+8 more
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Management of genetic diseases: Present and future
Today, the number of genetic diseases is around 10000 conditions, affecting to 6%-8% of all populations. This review shows us how the discovery of genetic variants in our genome, this facilitated to know with precision about the mechanisms ...
Hugo Hernán Abarca Barriga+2 more
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