Results 1 to 10 of about 7,531,202 (384)
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
Genetic therapy for pain management
Current Review of Pain, 2001 It has been suggested that there are two main approaches to the use of gene therapy in the treatment of chronic disease, which has been confirmed by recent studies in animals.
Steven P. Wilson, David C. Yeomans
openalex +6 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 ...
W. Bowers +2 more
semanticscholar +3 more sources
Targeted genetic repair: an emerging approach to genetic therapy [PDF]
Journal of Clinical Investigation, 2003 The last two decades have heralded a remarkable increase in our understanding of the genetic basis of disease and the endogenous mechanisms responsible for the repair of genomic DNA and the processing of RNA. Targeted gene repair is a powerful yet controversial technique developed to direct base changes in chromosomal genes, while RNA repair is an ...
Bruce A. Sullenger
openalex +4 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
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
openaire +4 more sources
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 +5 more sources