Application of Molecular Biology Studies to Gene Therapy Treatment Strategies

2002 ◽  
Vol 26 (7) ◽  
pp. 854-860 ◽  
Author(s):  
Allen Gustin ◽  
Lee Pederson ◽  
Ryan Miller ◽  
Carlos Chan ◽  
Selwyn M. Vickers
Keyword(s):  
Gene Therapy ◽  
Molecular Biology ◽  
Treatment Strategies ◽  
Therapy Treatment

Gene Therapy for Inherited Retinopathies: Update on Development Progress

2018 ◽  
Vol 2 (4) ◽  
pp. 219-226
Author(s):  
Susan Sun ◽  
Sandra R. Montezuma
Keyword(s):  
Gene Therapy ◽  
Genetic Disorders ◽  
Treatment Options ◽  
Genetic Material ◽  
Disease Process ◽  
Supportive Therapy ◽  
The Us ◽  
Retinal Disorders ◽  
The Status ◽  
Therapy Treatment

Inherited retinopathies are a group of genetic disorders that lead to blindness and/or vision impairment. Until now, treatment options for inherited retinopathies largely remained limited to supportive therapy. Gene therapy is an attractive therapeutic technique that allows repair of diseased genes, and it has shown success in vision improvement for patients affected by retinal disorders caused by genetic mutations. The US Food and Drug Administration approved the first gene therapy treatment for the eye, indicated for biallelic RPE65 mutation associated Leber congenital amaurosis (LCA), in December of 2017. Additionally, results from other ongoing clinical trials could further establish gene therapy as the milestone treatment that plays a role in disease process reversal for inherited retinopathies. This review article provides an update on the status of gene therapy for treatment of a variety of retinopathies, including LCA, choroideremia, achromatopsia, Stargardt disease, X-linked retinitis pigmentosa, and X-linked retinoschisis. Furthermore, this article explores transport methods of the genetic material, as well as therapy-delivery approaches used in the clinical setting.

scholarly journals Gene Therapy in the Management of Erectile Dysfunction (ED): Past, Present, and Future

2009 ◽  
Vol 9 ◽  
pp. 846-854 ◽  
Author(s):  
Arnold Melman ◽  
Kelvin P. Davies
Keyword(s):  
Gene Therapy ◽  
Erectile Dysfunction ◽  
Viral Vectors ◽  
Genetic Material ◽  
Phase I Trials ◽  
Clinical Phase ◽  
The Past ◽  
Novel Treatment ◽  
Therapy Treatment ◽  
Gene Transfer Techniques

In the past, many researchers considered viral vectors to be the most promising candidates to transfer genetic material into the corpora for the treatment of erectile dysfunction. However, at present, no viral vectors have progressed to human trials. In contrast, the use of naked gene therapy, a plasmid expressing the human Maxi-K potassium channel, is the only gene therapy treatment to be evaluated in clinical phase I trials to date. The success of these studies, proving the safety of this treatment, has paved the way for the development of future gene transfer techniques based on similar transfer methods, as well as novel treatment vectors, such as stem cell transfer.

Fundamentals of Molecular Biology and Gene Therapy

2010 ◽  
pp. 11-24
Author(s):  
Bert W. O’Malley ◽  
Daqing Li ◽  
Waleed M. Abuzeid ◽  
Hinrich Staecker
Keyword(s):  
Gene Therapy ◽  
Molecular Biology

scholarly journals Improving Precise CRISPR Genome Editing by Small Molecules: Is there a Magic Potion?

Cells ◽  
2020 ◽  
Vol 9 (5) ◽  
pp. 1318 ◽  
Author(s):  
Nadja Bischoff ◽  
Sandra Wimberger ◽  
Marcello Maresca ◽  
Cord Brakebusch
Keyword(s):  
Gene Therapy ◽  
Dna Repair ◽  
Animal Models ◽  
Molecular Biology ◽  
Small Molecules ◽  
Genome Editing ◽  
Drug Targets ◽  
Targeted Mutagenesis ◽  
Dna Repair Mechanisms ◽  
Repair Mechanisms

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing has become a standard method in molecular biology, for the establishment of genetically modified cellular and animal models, for the identification and validation of drug targets in animals, and is heavily tested for use in gene therapy of humans. While the efficiency of CRISPR mediated gene targeting is much higher than of classical targeted mutagenesis, the efficiency of CRISPR genome editing to introduce defined changes into the genome is still low. Overcoming this problem will have a great impact on the use of CRISPR genome editing in academic and industrial research and the clinic. This review will present efforts to achieve this goal by small molecules, which modify the DNA repair mechanisms to facilitate the precise alteration of the genome.

Molecular Biology of Neuroblastoma

1999 ◽  
Vol 17 (7) ◽  
pp. 2264-2264 ◽  
Author(s):  
John M. Maris ◽  
Katherine K. Matthay
Keyword(s):  
Molecular Biology ◽  
Susceptibility Gene ◽  
Genetic Material ◽  
Treatment Strategies ◽  
Specific Treatment ◽  
Chromosome Band ◽  
Suppressor Genes ◽  
Primary Tumors ◽  
Clinical Behavior ◽  
Expression Of Genes

PURPOSE AND RESULTS: Neuroblastoma, the most common solid extracranial neoplasm in children, is remarkable for its clinical heterogeneity. Complex patterns of genetic abnormalities interact to determine the clinical phenotype. The molecular biology of neuroblastoma is characterized by somatically acquired genetic events that lead to gene overexpression (oncogenes), gene inactivation (tumor suppressor genes), or alterations in gene expression. Amplification of the MYCN proto-oncogene occurs in 20% to 25% of neuroblastomas and is a reliable marker of aggressive clinical behavior. No other oncogene has been shown to be consistently mutated or overexpressed in neuroblastoma, although unbalanced translocations resulting in gain of genetic material from chromosome bands 17q23-qter have been identified in more than 50% of primary tumors. Some children have an inherited predisposition to develop neuroblastoma, but a familial neuroblastoma susceptibility gene has not yet been localized. Consistent areas of chromosomal loss, including chromosome band 1p36 in 30% to 35% of primary tumors, 11q23 in 44%, and 14q23-qter in 22%, may identify the location of neuroblastoma suppressor genes. Alterations in the expression of the neurotrophins and their receptors correlate with clinical behavior and may reflect the degree of neuroblastic differentiation before malignant transformation. Alterations in the expression of genes that regulate apoptosis also correlate with neuroblastoma behavior and may help to explain the phenomenon of spontaneous regression observed in a well-defined subset of patients. CONCLUSION: The molecular biology of neuroblastoma has led to a combined clinical and biologic risk stratification. Future advances may lead to more specific treatment strategies for children with neuroblastoma.

scholarly journals Development of Viral Vectors for Gene Therapy for Chronic Pain

2011 ◽  
Vol 2011 ◽  
pp. 1-8 ◽  
Author(s):  
Yu Huang ◽  
Xin Liu ◽  
Lanlan Dong ◽  
Zhongchun Liu ◽  
Xiaohua He ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Chronic Pain ◽  
Viral Vectors ◽  
Treatment Strategies ◽  
Opioid Analgesics ◽  
Cell Types ◽  
Health Concern ◽  
Specific Cell ◽  
New Treatment ◽  
The Individual

Chronic pain is a major health concern that affects millions of people. There are no adequate long-term therapies for chronic pain sufferers, leading to significant cost for both society and the individual. The most commonly used therapy for chronic pain is the application of opioid analgesics and nonsteroidal anti-inflammatory drugs, but these drugs can lead to addiction and may cause side effects. Further studies of the mechanisms of chronic pain have opened the way for development of new treatment strategies, one of which is gene therapy. The key to gene therapy is selecting safe and highly efficient gene delivery systems that can deliver therapeutic genes to overexpress or suppress relevant targets in specific cell types. Here we review several promising viral vectors that could be applied in gene transfer for the treatment of chronic pain and further discuss the possible mechanisms of genes of interest that could be delivered with viral vectors for the treatment of chronic pain.

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