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Preexisting Neutralizing Antibodies against Different Adeno-Associated Virus Serotypes in Humans and Large Animal Models for Gene Therapy
Gene therapy is a potential cure for several inherited retinal dystrophies, and adeno-associated virus (AAV) has emerged as a vector of choice for...
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Recent advances in various adeno-associated viruses (AAVs) as gene therapy agents in hepatocellular carcinoma
Primary liver cancer, which is scientifically referred to as hepatocellular carcinoma (HCC), is a significant concern in the field of global health....
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Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results
Limb-girdle muscular dystrophy 2E/R4 is caused by mutations in the β-sarcoglycan ( SGCB ) gene, leading to SGCB deficiency and consequent muscle loss....
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Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer’s disease
Adiponectin (APN) is an adipokine which predominantly expresses in adipocytes with neuroprotective and anti-inflammatory effects. We have recently...
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Suicide gene therapy using allogeneic adipose tissue-derived mesenchymal stem cell gene delivery vehicles in recurrent glioblastoma multiforme: a first-in-human, dose-escalation, phase I clinical trial
BackgroundGlioblastoma multiforme (GBM) is associated with remarkably poor prognosis, and its treatment is challenging. This investigation aimed to...
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Prospects for Nerve Regeneration and Gene Therapy in the Treatment of Traumatic Brain Injury
Traumatic brain injury (TBI) is a prevalent neurological disorder and a leading cause of death and disability worldwide. The high mortality rates...
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Progress in Intradermal and Transdermal Gene Therapy with Microneedles
Gene therapy is one of the most widely studied treatments and has the potential to treat a variety of intractable diseases. The skin's limited...
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Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair
Ex-vivo gene therapy has been shown to be an effective method for treating bone defects in pre-clinical models. As gene therapy is explored as a...
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Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort
Spinal muscular atrophy (SMA) is a motor neuron disease associated with progressive muscle weakness, ventilatory failure, and reduced survival....
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Long-term effects of a fat-directed FGF21 gene therapy in aged female mice
Fibroblast growth factor 21 (FGF21) has been developed as a potential therapeutic agent for metabolic syndromes. Moreover, FGF21 is considered a...
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Gene-Immune Therapy of Cancer: Approaches and Problems
AbstractTumor heterogeneity and constant selection of therapy-resistant cancer cells in the tumor require combinatorial approaches to the treatment...
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Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia
Many gene therapies are in development for treating people with inherited retinal diseases (IRD). We hypothesized that potential recipients of gene...
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Approaches to Gene Modulation Therapy for ALS
Amyotrophic lateral sclerosis (ALS) is a devastating motor neuron disease for which there is currently no robust therapy. Recent progress in...
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Non-viral approaches for gene therapy and therapeutic genome editing across the blood–brain barrier
The success of brain-targeted gene therapy and therapeutic genome editing hinges on the efficient delivery of biologics bypassing the blood–brain...
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Use of Mesenchymal Stem Cells to Enhance the Efficacy of Gene Therapy
Herein, a method to use of mesenchymal stem cells (MSCs) to modulate immune response against rAAV transduction in a canine Duchenne muscular...
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Transdermal delivery for gene therapy
Gene therapy is a critical constituent of treatment approaches for genetic diseases and has gained tremendous attention. Treating and preventing...
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Can gene therapy be used to prevent cancer? Gene therapy for aldehyde dehydrogenase 2 deficiency
Approximately 8% of the world population and 35–45% of East Asians are carriers of the hereditary disorder aldehyde dehydrogenase 2 (ALDH2)...
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Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency
Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34 + cell...
