scholarly journals Gene therapy: The ultimate cure for hereditary diseases

EBioMedicine ◽  
2019 ◽  
Vol 47 ◽  
pp. 1
Keyword(s):  
Gene Therapy ◽  
Hereditary Diseases ◽  
Ultimate Cure

Biotechnology

2021 ◽  
pp. 112-132
Author(s):  
Qing-Ping Ma
Keyword(s):  
Gene Therapy ◽  
Energy Consumption ◽  
Biological Systems ◽  
Hereditary Diseases ◽  
Future Trends ◽  
Current State ◽  
Ore Processing ◽  
Living Organisms ◽  
High Yields ◽  
Reducing Energy

Biotechnology utilizes biological systems or living organisms to create, develop, or make products. This chapter reviews the current state of biotechnology and examines its future trends. Currently, biotechnology plays key roles in medicine, agriculture, and industry. In medicine, vaccines which still rely on biological systems for their production, are the best tools to prevent infectious diseases; antibodies and RNA/DNA probes have been crucial in detecting and treating diseases; and genetic editing and gene therapy is making it possible to treat hereditary diseases. In agriculture, biotechnology is generating crops that produce high yields and need fewer inputs, crops that need fewer applications of pesticides, and crops with enhanced nutrition profiles. In industry, biotechnology is being utilized in food processing, metal ore processing, the production of chemicals, and reducing energy consumption and pollution.

Gene doping and genomic science in sports: where are we?

Bioanalysis ◽  
2020 ◽  
Author(s):  
Sheila López ◽  
João Meirelles ◽  
Vanessa Rayol ◽  
Gabriella Poralla ◽  
Nicole Woldmar ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Target Genes ◽  
Hereditary Diseases ◽  
Detection Methods ◽  
Sports Performance ◽  
Healthy Individuals ◽  
Control Field ◽  
Potential Health ◽  
Putative Target ◽  
World Anti Doping Agency

The misuse of sport-related gene transfer methods in elite athletes is a real and growing concern. The success of gene therapy in the treatment of hereditary diseases has been most evident since targets in gene therapy products can be used in healthy individuals to improve sports performance. Performing these practices threatens the sporting character of competitions and may pose potential health hazards. Since the World Anti-Doping Agency pronouncement on the prohibition of such practices in 2003, several researchers have been trying to address the challenge of developing an effective method for the detection of genetic doping. This review presents an overview of the published methods developed for this purpose, the advantages and limitations of technologies and the putative target genes. At last, we present the perspective related to the application of the detection methods in the doping control field.

scholarly journals TREATMENT POSSIBILITIES FOR ACQUIRED AND HEREDITARY DISEASES BY GENE THERAPY: A REVIEW

2021 ◽  
pp. 26-32
Author(s):  
P. V. KAMALA KUMARI ◽  
G. EKSHITHA, V. HARIKA
Keyword(s):  
Gene Therapy ◽  
Nucleic Acids ◽  
Viral Vectors ◽  
Target Gene ◽  
Genetic Diseases ◽  
Hereditary Diseases ◽  
Root Cause ◽  
Delivery Strategies ◽  
Foreign Genes ◽  
And Control

Therapeutic nucleic acids demand specificity and accuracy in design as well as delivery strategies used in replacement or silencing of the target gene. Gene therapy is believed to be the therapy in which the root cause of the diseases can be treated at the molecular level. Generally gene therapy helps in the identification of the origin of the disorder instead of using drugs to diminish or control the symptoms. The application of nucleic acids to treat and control diseases is known as “gene therapy.” Gene therapy consists on the substitution or addition of a functional gene into the nucleus of a living cell, in order to treat a disease or repair a dysfunction, caused by this gene failure. This therapy is used to correct defective genes, which are responsible for genetic diseases. Thus, gene therapy can be used to prevent, treat or regulate hereditary or acquired disorders, by the production of therapeutic proteins. The gene therapy is mediated by the use of viral and non-viral vectors to transport foreign genes into somatic cells to restorative defective genes. This review focuses on viral vectors in detail.

Evolutionary Timeline of Genetic Delivery and Gene Therapy

2020 ◽  
Vol 20 ◽  
Author(s):  
Natalie J. Holl ◽  
Han-Jung Lee ◽  
Yue-Wern Huang
Keyword(s):  
Gene Therapy ◽  
Effective Treatment ◽  
Small Molecule ◽  
Genetic Material ◽  
Elevated Risk ◽  
Hereditary Diseases ◽  
Physical Methods ◽  
Treatment Regimens ◽  
Small Molecule Drugs ◽  
Target Sites

: There are more than 3,500 genes that are being linked to hereditary diseases or correlated with elevated risk of certain illnesses. As an alternative to conventional treatments with small molecule drugs, gene therapy has arisen as an effective treatment with the potential to not just alleviate disease conditions but also cure them completely. In order for these treatment regimens to work, genes or editing tools intended to correct diseased genetic material must be efficiently delivered to target sites. There have been many techniques developed to achieve such a goal. In this article, we systematically review a variety of gene delivery and therapy methods that include physical methods, chemical and biochemical methods, viral methods, and genome editing. We discuss their historical discovery, mechanisms, advantages, limitations, safety, and perspectives.

scholarly journals Key achievements in gene therapy development and its promising progress with gene editing tools (ZFN, TALEN, CRISPR/Cas9)

2019 ◽  
Vol 2 (1) ◽  
pp. 1-9
Author(s):  
Dina Franic ◽  
Paula Dobrinic ◽  
Petra Korac
Keyword(s):  
Gene Therapy ◽  
Dna Sequences ◽  
Insertional Mutagenesis ◽  
Gene Editing ◽  
Hereditary Diseases ◽  
Target Tissue ◽  
Exogenous Dna ◽  
Therapy Development ◽  
The 1960S ◽  
Positive Results

Gene therapy concept is based on introduction of the wild-type allele into a patient’s genome in order to reverse a specific mutation. It is designed to treat hereditary diseases as well as the other diseases occurring later in life. Gene therapy was first mentioned in the 1960s and 70s, whereupon a series of studies was carried out, and in 1990 the first successful gene therapy was conducted. Since then about 2 600 clinical trials based on this concept were completed or are in progress. The two biggest issues are introduction of an exogenous DNA to target tissue, and its controlled integration in the genome. Until recently, the exogenous DNA sequences were incorporated randomly in the patient’s genome. Even though most of these treatments gave positive results, there was always a possibility of insertional mutagenesis. Controlling the integration place has rapidly progressed with the development of gene editing tools: ZFN, TALEN and CRISPR/Cas9. Although they have been used in only several clinical studies, gene editing tools are a small step away from clinical usage. In this review, we will give historical overview of gene therapy development and describe recent tools that can be used in precision medicine.

scholarly journals Amelioration of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integration

2021 ◽  
Author(s):  
Xi Chen ◽  
Xuran Niu ◽  
Yang Liu ◽  
Rui Zheng ◽  
Liren Wang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Specific Activity ◽  
High Specific Activity ◽  
Factor Ix ◽  
Hemophilia B ◽  
Hereditary Diseases ◽  
Proliferating Cells ◽  
Promising Therapeutic Approach ◽  
Targeted Integration

Site-specific integration of exogenous gene through genome editing is a promising strategy for gene therapy. However, homology-directed repair (HDR) only occurring in proliferating cells is inefficient especially in vivo. To investigate the efficacy of Cas9-induced homology-independent targeted integration (HITI) strategy for gene therapy, a rat hemophilia B model was generated and employed. Through HITI, a DNA sequence encoding the last exon of rat Albumin (rAlb) gene fused with a high-specific-activity Factor IX variant (R338L) using T2A, was inserted into the last intron of rAlb via recombinant adeno-associated viral (rAAV). The knock-in efficiency reached up to 3.66% determined by ddPCR. The clotting time was reduced to normal level 4 weeks after treatment, and the circulating FIX level was gradually increased up to 52% of normal during 9 months even after partial hepatectomy, demonstrating the amelioration of hemophilia. Through PEM-seq, no significant off-targeting effect was detected. Moreover, this study provides a promising therapeutic approach for hereditary diseases.

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