Genetic Therapy for Intervertebral Disc Degeneration [PDF]
International Journal of Molecular Sciences, 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 ...
Inbo Han +2 more
exaly +3 more sources
Genetic Therapy Approaches for Ornithine Transcarbamylase Deficiency. [PDF]
Biomedicines, 2023 Ornithine transcarbamylase deficiency (OTCD) is the most common urea cycle disorder with high unmet needs, as current dietary and medical treatments may not be sufficient to prevent hyperammonemic episodes, which can cause death or neurological sequelae.
Seker Yilmaz B, Gissen P.
europepmc +3 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
Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy. [PDF]
Mol Ther Nucleic Acids, 2021 Facioscapulohumeral muscular dystrophy (FSHD) is caused by chromatin relaxation of the D4Z4 repeat resulting in misexpression of the D4Z4-encoded DUX4 gene in skeletal muscle.
Šikrová D +5 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
Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders
Frontiers in Genetics, 2019 The field of gene therapy is striving more than ever to define a path to the clinic and the market. Twenty gene therapy products have already been approved and over two thousand human gene therapy clinical trials have been reported worldwide.
Saeed Mohammadi +2 more
exaly +2 more sources
Genetic Therapy and Molecular Targeted Therapy in Oncology: Safety, Pharmacovigilance, and Perspectives for Research and Clinical Practice [PDF]
International Journal of Molecular Sciences, 2022 Paolo Baldo +2 more
exaly +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
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 +3 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