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.

Cationic Carbosilane Dendrimers as Non‐viral Vectors of Nucleic Acids (Oligonucleotide or siRNA) for Gene Therapy Purposes

2013 ◽  
pp. 40-55 ◽  
Author(s):  
R. Gómez ◽  
F. J. de la Mata ◽  
J. L. Jiménez-Fuentes ◽  
P. Ortega ◽  
B. Klajnert ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Nucleic Acids ◽  
Viral Vectors ◽  
Carbosilane Dendrimers

scholarly journals Viral vectors and delivery strategies for CNS gene therapy

2010 ◽  
Vol 1 (4) ◽  
pp. 517-534 ◽  
Author(s):  
Steven J Gray ◽  
Kenton T Woodard ◽  
R Jude Samulski
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Delivery Strategies

scholarly journals Delivery Systems for Nucleic Acids and Proteins: Barriers, Cell Capture Pathways and Nanocarriers

Pharmaceutics ◽  
2021 ◽  
Vol 13 (3) ◽  
pp. 428
Author(s):  
Julian D. Torres-Vanegas ◽  
Juan C. Cruz ◽  
Luis H. Reyes
Keyword(s):  
Gene Therapy ◽  
Nucleic Acids ◽  
Genetic Diseases ◽  
Critical Factor ◽  
Cellular Internalization ◽  
Cell Capture ◽  
Inherited Disorders ◽  
Potential Approach ◽  
Efficient Delivery ◽  
Effective Delivery

Gene therapy has been used as a potential approach to address the diagnosis and treatment of genetic diseases and inherited disorders. In this line, non-viral systems have been exploited as promising alternatives for delivering therapeutic transgenes and proteins. In this review, we explored how biological barriers are effectively overcome by non-viral systems, usually nanoparticles, to reach an efficient delivery of cargoes. Furthermore, this review contributes to the understanding of several mechanisms of cellular internalization taken by nanoparticles. Because a critical factor for nanoparticles to do this relies on the ability to escape endosomes, researchers have dedicated much effort to address this issue using different nanocarriers. Here, we present an overview of the diversity of nanovehicles explored to reach an efficient and effective delivery of both nucleic acids and proteins. Finally, we introduced recent advances in the development of successful strategies to deliver cargoes.

scholarly journals Potential of helper-dependent Adenoviral vectors in CRISPR-cas9-mediated lung gene therapy

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Ranmal Avinash Bandara ◽  
Ziyan Rachel Chen ◽  
Jim Hu
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Lung Disease ◽  
Viral Vectors ◽  
Gene Editing ◽  
Genetic Diseases ◽  
Adenoviral Vectors ◽  
The Status

AbstractSince CRISPR/Cas9 was harnessed to edit DNA, the field of gene therapy has witnessed great advances in gene editing. New avenues were created for the treatment of diseases such as Cystic Fibrosis (CF). CF is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Despite the success of gene editing with the CRISPR/Cas9 in vitro, challenges still exist when using CRISPR/Cas9 in vivo to cure CF lung disease. The delivery of CRISPR/Cas9 into lungs, as well as the difficulty to achieve the efficiency required for clinical efficacy, has brought forth new challenges. Viral and non-viral vectors have been shown to deliver DNA successfully in vivo, but the sustained expression of CFTR was not adequate. Before the introduction of Helper-Dependent Adenoviral vectors (HD-Ad), clinical trials of treating pulmonary genetic diseases with first-generation viral vectors have shown limited efficacy. With the advantages of larger capacity and lower immunogenicity of HD-Ad, together with the versatility of the CRISPR/Cas9 system, delivering CRISPR/Cas9 to the airway with HD-Ad for lung gene therapy shows great potential. In this review, we discuss the status of the application of CRISPR/Cas9 in CF gene therapy, the existing challenges in the field, as well as new hurdles introduced by the presence of CRISPR/Cas9 in the lungs. Through the analysis of these challenges, we present the potential of CRISPR/Cas9-mediated lung gene therapy using HD-Ad vectors with Cystic Fibrosis lung disease as a model of therapy.

scholarly journals Innovative therapies in genetic diseases: Cystic fibrosis

2021 ◽  
Vol 70 (1) ◽  
pp. 16-20
Author(s):  
Elena-Silvia Shelby ◽  
◽  
Florina Mihaela Nedelea ◽  
Tanser Huseyinoglu ◽  
Relu Cocos ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Clinical Trials ◽  
Antisense Oligonucleotides ◽  
Viral Vectors ◽  
Genetic Diseases ◽  
Wild Type ◽  
Gene Encoding ◽  
Innovative Therapies ◽  
Type Gene

Cystic fibrosis, also named mucoviscidosis, is the most frequent hereditary pulmonary disease and is produced by mutations in the CFTR gene, encoding an anionic channel for chloride and bicarbonate involved in the regulation of salt and bicarbonate metabolisms. Currently, about half of the patients with cystic fibrosis can benefit personalized therapy consisting in modulators, drugs which restore or improve the functionality and stability of CFTR. Moreover, presently, other therapies, such as gene therapy using the CRISP/CAS-9, modified antisense oligonucleotides or the insertion of the wild-type gene using nanolipidic particles or viral vectors, are being developed. This article aims to take stock of the principal types of cystic fibrosis therapies which have been approved or are in clinical trials.

scholarly journals Muscle-Specific Promoters for Gene Therapy

Acta Naturae ◽  
2021 ◽  
Vol 13 (1) ◽  
pp. 47-58
Author(s):  
Victoria V. Skopenkova ◽  
Tatiana V. Egorova ◽  
Maryana V. Bardina
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Genetic Diseases ◽  
Gene Replacement ◽  
Regulatory Sequences ◽  
Muscle Creatine Kinase ◽  
Specific Expression ◽  
Drug Candidates ◽  
Muscular Disorders ◽  
Preclinical Trials

Many genetic diseases that are responsible for muscular disorders have been described to date. Gene replacement therapy is a state-of-the-art strategy used to treat such diseases. In this approach, the functional copy of a gene is delivered to the affected tissues using viral vectors. There is an urgent need for the design of short, regulatory sequences that would drive a high and robust expression of a therapeutic transgene in skeletal muscles, the diaphragm, and the heart, while exhibiting limited activity in non-target tissues. This review focuses on the development and improvement of muscle-specific promoters based on skeletal muscle -actin, muscle creatine kinase, and desmin genes, as well as other genes expressed in muscles. The current approaches used to engineer synthetic muscle-specific promoters are described. Other elements of the viral vectors that contribute to tissue-specific expression are also discussed. A special feature of this review is the presence of up-to-date information on the clinical and preclinical trials of gene therapy drug candidates that utilize muscle-specific promoters.

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