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Nanoplatform‐Enabled Genetic Interventions for Central Nervous System Disorders: Advances in Delivery Strategies and Therapeutic Potential [PDF]

open access: yesAdvanced Genetics
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
doaj   +2 more sources

Biomimetic Peptides: A New Generation of Gene Transfer Vectors [PDF]

open access: yesمجله 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
doaj   +1 more source

A review of Genetic Etiology and Emerging Molecular Therapies for FSHD in Preclinical Studies [PDF]

open access: yesReviews 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
doaj   +1 more source

Differentiation of Adipose-Derived Mesenchymal Stem Cells into Neuron-Like Cells induced by using β-mercaptoethanol

open access: yesمجلة 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
doaj   +1 more source

Interleukins, growth factors, and transcription factors are key targets for gene therapy in osteoarthritis: A scoping review

open access: yesFrontiers 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
doaj   +1 more source

Human Neural Stem Cells: Translational Research for Neonatal Hypoxic-Ischemic Brain Injury [PDF]

open access: yesNeonatal 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
doaj   +1 more source

The First Homozygote Mutation c.499G>T (Asp167Tyr) in the RPE65 Gene Encoding Retinoid Isomerohydrolase Causing Retinal Dystrophy

open access: yesCurrent Issues in Molecular Biology, 2022
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
doaj   +1 more source

DNA Edition: ad portas of a revolution in genetic manipulation

open access: yesIatreia, 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
doaj   +1 more source

Translating the combination of gene therapy and tissue engineering for treating recessive dystrophic epidermolysis bullosa [PDF]

open access: yesEuropean Cells & Materials, 2018
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
doaj   +1 more source

Management of genetic diseases: Present and future

open access: yesRevista de la Facultad de Medicina Humana, 2021
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
doaj   +1 more source

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