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The estimated annual financial impact of gene therapy in the United States
Gene therapy is a new class of medical treatment that alters part of a patient’s genome through the replacement, deletion, or insertion of genetic...
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Txnip Gene Therapy of Retinitis Pigmentosa Improves Cone Health
Retinitis pigmentosa (RP) is a hereditary retinal degenerative disease that can lead to blindness. In RP, rod photoreceptors die first, followed by...
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Artificial Intelligence and Computational Biology in Gene Therapy: A Review
One of the trending fields in almost all areas of science and technology is artificial intelligence. Computational biology and artificial...
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Gene Therapy Strategies for Prophylactic and Therapeutic Treatments of Human Prion Diseases
Prion disease is a diverse family of fatal and usually transmissible and progressive neurodegenerative diseases that strike humans and many other...
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A Brief Introduction to Current Cancer Gene Therapy
Gene therapy has started in the late 1980s as novel, clinically applicable therapeutic option. It revolutionized the treatment of genetic diseases...
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AAV-based in vivo gene therapy for neurological disorders
Recent advancements in gene supplementation therapy are expanding the options for the treatment of neurological disorders. Among the available...
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Assessment of the Gene Therapy Immune Response in the Canine Muscular Dystrophy Model
The immune response is a primary hurdle in the development of gene therapy for neuromuscular diseases. Both innate and adaptive immune responses have...
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MRI Evaluation of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy
Magnetic resonance imaging (MRI) is a well-established and widely used technique to characterize and quantify skeletal and cardiac muscle changes in...
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Viral Vector-Based Gene Therapy for Epilepsy: What Does the Future Hold?
In recent years, many pre-clinical studies have tested gene therapy approaches as possible treatments for epilepsy, following the idea that they may...
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Novel Gene Therapy Approaches for Targeting Neurodegenerative Disorders: Focusing on Delivering Neurotrophic Genes
Neurodegenerative illnesses (NDDs) like Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis, spinal muscular atrophy, and Huntington’s disease...
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Retroviral Vectors for Gene Therapy of Monogenic Diseases
Gene therapy vectors derived from retroviruses are attractive for treating monogenic diseases due to the stable integration of a transgene in patient...
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Acoustically targeted noninvasive gene therapy in large brain volumes
Focused Ultrasound Blood-Brain Barrier Opening (FUS-BBBO) can deliver adeno-associated viral vectors (AAVs) to treat genetic disorders of the brain....
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Challenges of Treatment Methodologies and the Future of Gene Therapy and Stem Cell Therapy to Treat Retinitis Pigmentosa
Retinitis pigmentosa (RP) is a heterogeneous group of hereditary retinal degenerations for which there is currently no cure. Studies investigating...
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In vivo and ex vivo gene therapy for neurodegenerative diseases: a promise for disease modification
Neurodegenerative diseases (NDDs), including AD, PD, HD, and ALS, represent a growing public health concern linked to aging and lifestyle factors,...
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Progress in Clinical Gene Therapy for Cardiac Disorders
Despite significant advances in novel treatments and approaches, cardiovascular disease remains the leading cause of death globally. Gene therapy is...
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Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results
Gene therapy is a promising approach for hereditary deafness. We recently showed that unilateral AAV1-hOTOF gene therapy with dual adeno-associated...
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Stem Cells and Targeted Gene Therapy in Brain and Spinal Cord Tumors
The CNS tumors, in particular those with malignantMalignant characteristics, are prominent burdens around the world with high mortalityMortality and...
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Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa
Somatic gene therapy will be one of the most exciting practices of genetic medicine in Africa and is primed to offer a “new life” for persons living...
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TCR Gene Therapy for Cancer
The protocol describes the procedure of antigen-specific T cellT-cell generation and TCR T-cell receptor (TCR) identification for the use in...
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Intravitreal Delivery of PEGylated-ECO Plasmid DNA Nanoparticles for Gene Therapy of Stargardt Disease
ObjectiveCurrent gene therapy of inherited retinal diseases is achieved mainly by subretinal injection, which is invasive with severe adverse...
