Exosomes as promising gene therapy tools still need to be researched and manufactured more efficiently

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Gene Interactions ◽  
Target Cells ◽  
Related Gene ◽  
Significant Progress ◽  
Delivery Methods ◽  
P Gene ◽  
Great Progress ◽  
Mode Of Transportation ◽  
Disease Related Gene

Gene therapy was first established in 1972, and since then, it has made great progress in terms of adoption. The efficiency of the gene transfer carrier is critical to the success of gene therapy. As a result, researchers are always looking for a secure, specialized, and efficient mode of transportation. In recent years, the newly discovered exosomal transport mechanism has made significant progress. Viruses, bacteria, phages, and synthetic lipids-based delivery methods are less stable, biocompatible, and can cross the blood-brain barrier. The majority of the research has focused on creating exosomes that transport therapeutic miRNA to specific target cells. Despite the excellent results, there are still some concerns about employing exosomes as a gene therapy carrier. The miRNA mechanism is becoming increasingly apparent for a specific condition, yet one miRNA can influence several genes. Furthermore, nothing is known about numerous non-disease related gene interactions. Although high levels of certain exocrine miRNA may aid in the treatment of some disorders, nothing is known about potential side effects. Exosomes still need to be researched and manufactured more efficiently. While exosomes have significant promise for gene therapy, using exocrine miRNAs to treat disorders is difficult and requires further research and development by academics and clinicians.

Potential strategies for cancer gene therapy

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Clinical Stage ◽  
Genetic Material ◽  
Direct Methods ◽  
Cancer Gene Therapy ◽  
Target Cells ◽  
Target Tissue ◽  
Cancer Gene ◽  
P Gene

Gene therapy involves transferring genetic material (DNA or RNA) to repair, regulate or replace genes to cure a disease. One of the most crucial barriers is successful delivery of the targeted gene into the target tissue. Various vector-based approaches have been developed to deliver the transgene to the target cells. In different cancers, numerous of these vectors are being developed for purposes such as immunotherapy, suicide gene therapy, microRNA (miRNA) focused treatment, oncogene silencing, and gene editing using CRISPR/Cas9. This article reviews several alternatives to cancer gene therapy, as well as their preclinical and clinical outcomes, possible limitations, and overall therapy effects. Ways of delivering cancer gene therapy include direct methods for introducing genetic material. Nonviral vectors are easy to manufacture and may be chemically modified to increase their usefulness. Cationic polymers such as Poly-L-Lysine (PLL) and Polyethylenimine (PEI-SS) are the most extensively used polycationic polymers for gene transfer, particularly in vitro. Many RNAi-based therapeutic approaches are approaching the clinical stage, and nanocarriers are likely to play a crucial role in treating specific cancers. In the previous decade, non-viral approaches were used in more than 17 percent of all gene therapy trials. The message is that this is a safe and effective technique for transferring genes to cancer patients who need it to be a safe, successful therapy. Exosomes were developed to carry oncogene-specific short interfering RNA. Sushrut and colleagues revealed that exosomes provide superior carriers of short RNA and prevent tumor growth than liposomes. Inhalation-based gene therapy (aerosol-mediated gene delivery) has gained pace as a feasible treatment approach, especially for lung cancer. Because the intended transgene is steered to specific cells/tissues, this should further increase therapeutic efficiency.

Substance P Containing Peptide Gene Delivery Vectors for Specifically Transfecting Glioma Cells Mediated by Neurokinin-1 Receptor

2021 ◽  
Author(s):  
Guihua Ding ◽  
Taoran Wang ◽  
Zhenbin Han ◽  
Long Tian ◽  
Qin Cheng ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glioblastoma Multiforme ◽  
Substance P ◽  
Glioma Cells ◽  
Target Cells ◽  
P Gene ◽  
Gene Delivery Vectors ◽  
Promising Treatment ◽  
Neurokinin 1 ◽  
Peptide Gene

Gene therapy provides a promising treatment for glioblastoma multiforme, which mainly depends on two keys, crossing the blood brain barrier (BBB) effectively and transfecting target cells selectively. In this work,...

Viral Vector Delivery of DREADDs for CNS Therapy

2021 ◽  
Vol 21 ◽  
Author(s):  
Ceri A. Pickering ◽  
Nicholas D. Mazarakis
Keyword(s):  
Gene Therapy ◽  
Viral Vector ◽  
Designer Drugs ◽  
Target Cells ◽  
Delivery Methods ◽  
Therapy Strategy ◽  
Synthetic Ligand ◽  
The Central Nervous System ◽  
Gene Therapy Strategy ◽  
Vector Delivery

: Designer Receptors Exclusively Activated by Designer Drugs (DREADDs) are genetically modified G-protein-coupled receptors (GPCRs), which can be activated by a synthetic ligand that is otherwise inert at endogenous receptors. DREADDs can be expressed in cells in the central nervous system (CNS) and subsequently offer the opportunity for remote and reversible silencing or activation of the target cells when the synthetic ligand is systemically administered. In neuroscience, DREADDs have thus far shown to be useful tools for several areas of research. Furthermore, they offer considerable potential for use as a gene therapy strategy for neurological disorders. However, in order to design a DREADD-based gene therapy, it is necessary to first evaluate the viral vector delivery methods utilised to deliver these chemogenetic tools in the literature. This review evaluates each of the prominent strategies currently utilised for DREADD delivery, discussing their respective advantages and limitations. It focuses on Adeno-Associated Virus (AAV)- and lentivirus-based systems, and the manipulation of these through cell-type specific promoters and pseudotyping. Furthermore, we address how virally mediated DREADD delivery could be improved in order to make it a viable gene therapy strategy and thus expand its translational potential.

Non-Viral Vectors for Gene Delivery

2018 ◽  
Vol 9 (1) ◽  
pp. 4-11 ◽  
Author(s):  
Aparna Bansal ◽  
Himanshu
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Transfection Efficiency ◽  
Gene Transfection ◽  
Expression System ◽  
Target Cells ◽  
Specific Expression ◽  
Naked Dna

Introduction: Gene therapy has emerged out as a promising therapeutic pave for the treatment of genetic and acquired diseases. Gene transfection into target cells using naked DNA is a simple and safe approach which has been further improved by combining vectors or gene carriers. Both viral and non-viral approaches have achieved a milestone to establish this technique, but non-viral approaches have attained a significant attention because of their favourable properties like less immunotoxicity and biosafety, easy to produce with versatile surface modifications, etc. Literature is rich in evidences which revealed that undoubtedly, non–viral vectors have acquired a unique place in gene therapy but still there are number of challenges which are to be overcome to increase their effectiveness and prove them ideal gene vectors. Conclusion: To date, tissue specific expression, long lasting gene expression system, enhanced gene transfection efficiency has been achieved with improvement in delivery methods using non-viral vectors. This review mainly summarizes the various physical and chemical methods for gene transfer in vitro and in vivo.

Transcriptional impact of resminostat and other HDAC inhibitors on disease-related gene expression in CTCL

2019 ◽  
Vol 119 ◽  
pp. S10
Author(s):  
A.C. Bretz ◽  
G. Streubel ◽  
U. Parnitzke ◽  
M. Borgmann ◽  
S. Hamm
Keyword(s):  
Gene Expression ◽  
Hdac Inhibitors ◽  
Related Gene ◽  
Disease Related Gene

Regulation of gene transfection by cell size, shape and elongation on micropatterned surfaces

2021 ◽  
Author(s):  
Yongtao Wang ◽  
Yingjun Yang ◽  
Toru Yoshitomi ◽  
Naoki Kawazoe ◽  
Yingnan Yang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Cell Size ◽  
Tissue Repair ◽  
Gene Transfection ◽  
P Gene ◽  
Gene Carriers ◽  
Potential Applications

Gene transfection has been widely studied due to its potential applications in tissue repair and gene therapy. Many studies have focused on designing gene carriers and developing novel transfection techniques....

scholarly journals Cryopreservation of microglia enables single-cell RNA sequencing with minimal effects on disease-related gene expression patterns

iScience ◽  
2021 ◽  
Vol 24 (4) ◽  
pp. 102357
Author(s):  
Brenda Morsey ◽  
Meng Niu ◽  
Shetty Ravi Dyavar ◽  
Courtney V. Fletcher ◽  
Benjamin G. Lamberty ◽  
...  
Keyword(s):  
Gene Expression ◽  
Single Cell ◽  
Rna Sequencing ◽  
Expression Patterns ◽  
Gene Expression Patterns ◽  
Related Gene ◽  
Single Cell Rna Sequencing ◽  
Disease Related Gene

Thyroid Hormone Up-Regulates an Alzheimer Disease–Related Gene Seladin-1 at the Transcriptional Level

2011 ◽  
pp. OR30-3-OR30-3 ◽  
Author(s):  
Emi Ishida ◽  
Koshi Hashimoto ◽  
Atsushi Ozawa ◽  
Nobuyuki Shibusawa ◽  
Tetsurou Satoh ◽  
...  
Keyword(s):  
Thyroid Hormone ◽  
Alzheimer Disease ◽  
Transcriptional Level ◽  
Related Gene ◽  
Disease Related Gene
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