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Nanodelivery in Gene Therapy
Gene therapy stands as a revolutionary approach in medicine, aiming to rectify genetic abnormalities by introducing therapeutic genes or regulating...
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Gene Therapy and Cardiovascular Diseases
Cardiovascular diseases (CVDs) are the leading causes of death globally and urgently require new novel therapeutic strategies. Gene therapy is the...
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Gene Therapy in Molecular Biology and Drug Delivery
The objective of gene therapy is to manipulate/modify the gene expression or modify living cell characteristics for therapeutic utilization. It aims...
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Immune Gene Therapy of Cancer
Cancer is the second global cause of mortality. Conventional treatments, such as chemotherapy and radiotherapy, have not provided desirable...
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Gene Therapy of Cancer Methods and Protocols
This third edition provides new and updated chapters on gene therapeutic strategies of cancer. Chapters guide readers through suicide and oncolytic...
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Prospective approaches to gene therapy computational modeling – spotlight on viral gene therapy
Clinical studies have found there still exists a lack of gene therapy dose-toxicity and dose-efficacy data that causes gene therapy dose selection to...
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Gene Therapy of Extracellular Vesicles in Cardiovascular and Metabolic Diseases
The ultimate and most complex form of treating human diseases is embodied by gene therapy. For an effective gene therapeutic product we need to hack...
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Microfluidic Approaches for Gene Delivery and Therapy
Gene editing techniques such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/Cas9) have been developed to target a gene into...
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AAV Serotypes and Their Suitability for Retinal Gene Therapy
Throughout the last 25 years, exceptional progress in retinal gene therapy was achieved. The major breakthrough was realized in 2017 when the FDA...
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Biotechnologies for Gene Therapy RNA, CRISPR, Nanobots, and Preclinical Applications
The purpose of this book is to highlight some of latest developments and applications of CRISPR, RNA, and DNA to treat diseases ranging from cancers...
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Phase-Appropriate Potency Assay Development for AAV-Based Gene Therapy Products
The development of any medicinal product requires thorough characterization and quality control to assure drug product potency. Adeno-associated...
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Cancer Gene Therapy: Development and Production of Lentiviral Vectors for Gene Therapy
Lentiviral vectors are among the most used vectors in gene therapy to treat pathologies of different origins, such as cancers, rare monogenic...
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Gene therapy for CNS disorders: modalities, delivery and translational challenges
Gene therapy is emerging as a powerful tool to modulate abnormal gene expression, a hallmark of most CNS disorders. The transformative potentials of...
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Surgical Approach with Pars Plana Vitrectomy for Subretinal Gene Therapy
Gene therapy is emerging as a treatment for inherited diseases including retinitis pigmentosa. Through surgery, specifically with pars plana...
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A multinational survey of potential participant perspectives on ocular gene therapy
Amidst rapid advancements in ocular gene therapy, understanding patient perspectives is crucial for sha** future treatment choices and research...
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Successes and challenges in clinical gene therapy
Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provide enduring treatments for...
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Gene therapy: an alternative to treat Alzheimer’s disease
Alzheimer’s disease (AD), a neuro-degenerative disease that primarily affects the elderly, is a worldwide phenomenon. Loss of memory, cognitive...
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Ewing sarcoma treatment: a gene therapy approach
Ewing sarcoma (ES) is an aggressive malignant tumor, characterized by non-random chromosomal translocations that produce fusion genes. Fusion genes...
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Deafness: from genetic architecture to gene therapy
Progress in deciphering the genetic architecture of human sensorineural hearing impairment (SNHI) or loss, and multidisciplinary studies of mouse...
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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...