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Gene therapy: principles, challenges and use in clinical practice
IntroductionGene therapy is an emerging topic in medicine. The first products have already been licensed in the European Union for the treatment of...
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Systematic gene therapy derived from an investigative study of AAV2/8 vector gene therapy for Fabry disease
BackgroundFabry disease (FD) is a progressive multisystemic disease characterized by a lysosomal enzyme deficiency. A lack of α-galactosidase A...
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Cell-Based Gene Therapy for β-Thalassemia
The United States Food and Drug Administration (FDA) approved betibeglogene autotemcel (beti-cel), the first cell-based gene therapy for adult and...
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Cell and gene therapy for kidney disease
Kidney disease is a leading cause of morbidity and mortality across the globe. Current interventions for kidney disease include dialysis and renal...
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Application of Gene Therapy in Hemophilia
Gene therapy refers to introducing normal exogenous genes into target cells to correct or compensate for the diseases caused by defective and...
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Adeno-associated virus as a delivery vector for gene therapy of human diseases
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to...
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Hepatotoxicity in Adeno-Associated Viral Vector Gene Therapy
Purpose of ReviewRecombinant adeno-associated virus (rAAV) is the commonest viral vector used in gene therapies. With the increase in the number of...
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Gene therapy for liver diseases — progress and challenges
Gene therapy is poised to revolutionize modern medicine, with seemingly unlimited potential for treating and curing genetic disorders. For otherwise...
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Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study
BackgroundGene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment...
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Gene Therapy for Neurofibromatosis Type 2-Related Schwannomatosis: Recent Progress, Challenges, and Future Directions
Neurofibromatosis type 2 ( NF2 )-related schwannomatosis is a rare autosomal dominant monogenic disorder caused by mutations in the NF2 gene. The...
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Liposomal delivery of gene therapy for ovarian cancer: a systematic review
ObjectiveTo systematically identify and narratively synthesize the evidence surrounding liposomal delivery of gene therapy and the outcome for...
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Retinal gene therapy in RPE-65 gene mediated inherited retinal dystrophy
BackgroundVoretigene neparvovec (VN) is a gene therapeutic agent for treatment of retinal dystrophies caused by bi-allelic RPE65 mutations. We...
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Taking the knife to neurodegeneration: a review of surgical gene therapy delivery to the CNS
Gene supplementation and editing for neurodegenerative disorders has emerged in recent years as the understanding of the genetic mechanisms...
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Gene Therapy for Inherited Retinal Diseases: From Laboratory Bench to Patient Bedside and Beyond
This comprehensive review provides a thorough examination of inherited retinal diseases (IRDs), encompassing their classification, genetic...
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Viral-mediated gene therapy in pediatric neurological disorders
BackgroundDue to the broad application of next-generation sequencing, the molecular diagnosis of genetic disorders in pediatric neurology is no...
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A position statement on the post gene-therapy rehabilitation of aromatic I-amino acid decarboxylase deficiency patients
Aromatic l -amino acid decarboxylase (AADC) deficiency is a rare genetic disorder of monoamine neurotransmitter synthesis that presents with a range...
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Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective
Onasemnogene abeparvovec has been life-changing for children with spinal muscular atrophy (SMA), signifying the potential and progress occurring in...
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X-Linked Retinitis Pigmentosa Gene Therapy: Preclinical Aspects
The most common inherited eye disease is retinitis pigmentosa (RP). X-linked RP (XLRP) is one of the most severe types of RP, with a considerable...
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Ex vivo dual gene therapy using human adipocytes secreting anti-HER2 antibody on HER2-positive xenograft tumor models
BackgroundAlthough targeted treatments against human epidermal growth factor receptor 2 (HER2) have improved survival in patients with metastatic...
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Gene Therapy for Congenital Hearing Loss
Purpose of ReviewThis review provides the otolaryngology community with background on the basics of gene therapy for congenital hearing loss along...
