Results 1 to 10 of about 8,660,824 (403)
Genetic Therapy for Intervertebral Disc Degeneration. [PDF]
Int J Mol Sci, 2021 Intervertebral disc (IVD) degeneration can cause chronic lower back pain (LBP), leading to disability. Despite significant advances in the treatment of discogenic LBP, the limitations of current treatments have sparked interest in biological approaches ...
Roh EJ +7 more
europepmc +2 more sources
Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models. [PDF]
Gene Ther, 2022 Spinal muscular atrophy (SMA) is a severe childhood neuromuscular disease for which two genetic therapies, Nusinersen (Spinraza, an antisense oligonucleotide), and AVXS-101 (Zolgensma, an adeno-associated viral vector of serotype 9 AAV9), have recently ...
Chilcott EM +3 more
europepmc +2 more sources
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.
Theodore L. Roth, A. Marson
semanticscholar +5 more sources
Dis Model Mech, 2016 Hypomyelination is a key symptom of Allan-Herndon-Dudley syndrome (AHDS), a psychomotor retardation associated with mutations in the thyroid-hormone (TH) transporter MCT8 (monocarboxylate transporter 8).
Zada D +3 more
europepmc +2 more sources
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
doaj +1 more source
Deafness: from genetic architecture to gene therapy
Nature reviews genetics, 2023 Progress in deciphering the genetic architecture of human sensorineural hearing impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the elucidation of the molecular mechanisms underlying auditory system function ...
C. Petit, C. Bonnet, S. Safieddine
semanticscholar +1 more source
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
doaj +1 more source
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
openaire +4 more sources
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
doaj +1 more source
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
doaj +1 more source