Results 11 to 20 of about 8,605,496 (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
Genetic therapy for the nervous system [PDF]
Human Molecular Genetics, 2011 Genetic therapy is undergoing a renaissance with expansion of viral and synthetic vectors, use of oligonucleotides (RNA and DNA) and sequence-targeted regulatory molecules, as well as genetically modified cells, including induced pluripotent stem cells from the patients themselves. Several clinical trials for neurologic syndromes appear quite promising.
William J, Bowers +2 more
openaire +3 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
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
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
doaj +1 more source
Current 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
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
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2022 Adeno-associated virus (AAV) vectors are promising modalities of gene therapy to address unmet medical needs. However, anti-AAV neutralizing antibodies (NAbs) hamper the vector-mediated therapeutic effect.
Yuji Kashiwakura +29 more
doaj +1 more source
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
openaire +2 more sources