RNA Nanoparticles for Therapy of Cancer, Viral Infections, and Genetic Diseases

2013 ◽  
pp. 361-362
Biomeditsina ◽  
2019 ◽  
pp. 34-42
Author(s):  
A. A. Mokhov ◽  
A. A. Chaplenko ◽  
A. N. Yavorskiy

Genome editing technologies are currently based on the use of one from the three classes of nucleases, i.e. a zinc finger, TAL or CRISPR-Cas. Drawbacks inherent in each of these approaches, though not being critical for animal or in vitro experiments, significantly limit their application in human genome editing. Considerable experience has so far been accumulated in the field of using gene-editing technologies for the treatment and prevention of genetic diseases, transmissible and viral infections. However, further progress is hampered by various technical and ethical problems. It is the task of expert communities and the state that genomic editing methods be smoothly integrated into everyday practices without significant social upheavals.


2006 ◽  
Vol 10 (13) ◽  
pp. 88-155

SCBA Special WORKSHOP #1 NIH WORKSHOP NIH — Goes Electronic: How to Get Grants In the E-age? SCBA Special WORKSHOP #2 ACGA WORKSHOP — Genetic Diseases and Genomic Medicine. SCBA Special WORKSHOP #3 Genetic Changes and Mechanisms Contributing to Human Cancer. SCBA Special WORKSHOP #4 Upward Mobility in the Industrial and Academic Career. SCBA WORKSHOP #1: RNA Interference. SCBA WORKSHOP #2: Vaccine and Antiviral Therapies. SCBA WORKSHOP #3: Regulation and Modification of p53. SCBA WORKSHOP #4: Opioid Receptors: Molecules, Cells and the Whole Animal. SCBA WORKSHOP #5: Nuclear Receptor. SCBA WORKSHOP #6: Immune Responses and Signaling. SCBA WORKSHOP #7: Frontiers in Gene Therapy. SCBA WORKSHOP #8: Pathogenesis and Treatments of Neurophsychiatric and Neurodegenerative Diseases: New Understandings and New Possibilities. SCBA WORKSHOP #9: Nanotechnologies and Microfluidics for Biotech Application. SCBA WORKSHOP #10: Basic Mechanisms of Ubiquitination, NEDDDylation ISG-15 Modification. SCBA WORKSHOP #11: Neuronal Signaling and Synaptic Plasticity. SCBA WORKSHOP #12: Signal Transduction 1. SCBA WORKSHOP #13: Chemical Genetics. SCBA WORKSHOP #14: Plant Science and Epigenics Biology. SCBA WORKSHOP #15: Cytokines and Inflammation. SCBA WORKSHOP #16: Novel Post-Translation Modifications. SCBA WORKSHOP #17: Translational Medicine. SCBA WORKSHOP #18: Acetylationa in Ubiquitinatio in Chromatin-Templated Processes. SCBA WORKSHOP #19: Pathogenesis of Viral Infections. SCBA WORKSHOP #20: Stem Cell Biology and Regenerative Medicine. SCBA WORKSHOP #21: Biotech Panel: The making of a Successful Biotech Company. SCBA WORKSHOP #22: Molecular Mechanisms of Cancer Progression and Therapeutic Resistance. SCBA WORKSHOP #23: Regulatory Geneomics and Epigenomics. SCBA WORKSHOP #24: RNA Biology. SCBA WORKSHOP #25: Bioprocessing. SCBA WORKSHOP #26: Recent Development of Targeted Therapy. SCBA WORKSHOP #27: DNA Damage Response in Eukaryotes. SCBA WORKSHOP #28: The Biology and Promise of Neural Stem Cells. SCBA WORKSHOP #29: IP and Business Licensing. SCBA WORKSHOP #30: Basic Mechanisms of Sumoylation. SCBA WORKSHOP #31: Immune Regulation and Therapy. SCBA WORKSHOP #32: Proteomics and Applications.


2018 ◽  
Vol 20 (3) ◽  
pp. 177 ◽  
Author(s):  
A.S. Levina ◽  
M.N. Repkova ◽  
Z.R. Ismagilov ◽  
V.F. Zarytova

A promising new approach to the treatment of viral infections and genetic diseases associated with damaged or foreign nucleic acids in the body is gene therapy, i.e., the use of antisense oligonucleotides, ribozymes, deoxyribozymes, siRNA, plasmid DNA, etc. (therapeutic nucleic acids). Selective recognition of target nucleic acids by these compounds based on highly specific complementary interaction can minimize negative side effects, which occur with currently used low molecular weight drugs. To apply a new generation of therapeutic agents in medical practice, it is necessary to solve the problem of their delivery into cells. Silicon-containing nanoparticles are considered as promising carriers for this purpose due to their biocompatibility, low toxicity, ability to biodegradation and excretion from the body, as well as the simplicity of the synthesis and modification. Silicon-containing nanoparticles are divided into two broad categories: solid (nonporous) and mesoporous silicon nanoparticles (MSN). This review gives a brief overview of the creation of mesoporous, multilayer, and other silicon-based nanoparticles. The publications concerning solid silicon-organic nanoparticles capable of binding and delivering nucleic acids into cells are discussed in more detail with emphasis on methods for their synthesis. The review covers publications over the past 15 years, which describe the classical Stöber method, the microemulsion method, modification of commercial silica nanoparticles, and other strategies.


2021 ◽  
Vol 8 (1) ◽  
pp. 115-128
Author(s):  
A. A. Chaplenko ◽  
A. A. Mokhov ◽  
A. N. Yavorsky

Significant progress has been made in the development of genetic technologies in recent decades. Currently, high-performance sequencing and, most importantly, genome editing technologies are widely used and available for laboratories in Russia. Existing technologies are not without drawbacks that significantly hinder further development, in addition, all the necessary reagents and materials, as well as equipment, are produced exclusively abroad. The review highlights the international experience of using genome editing technologies for the treatment and prevention of genetic diseases, vector-borne and viral infections, it offers recommendations for the development of this area in the Russian Federation. Attention is drawn to the legal and ethical regulation, mainly at the level of basic principles. The conclusion is formulated on the need for accelerated adaptation of basic ethical and legal principles for genome editing activities in scientific biomedical activities.


Author(s):  
William B. McCombs ◽  
Cameron E. McCoy

Recent years have brought a reversal in the attitude of the medical profession toward the diagnosis of viral infections. Identification of bacterial pathogens was formerly thought to be faster than identification of viral pathogens. Viral identification was dismissed as being of academic interest or for confirming the presence of an epidemic, because the patient would recover or die before this could be accomplished. In the past 10 years, the goal of virologists has been to present the clinician with a viral identification in a matter of hours. This fast diagnosis has the potential for shortening the patient's hospital stay and preventing the administering of toxic and/or expensive antibiotics of no benefit to the patient.


Author(s):  
S. E. Miller ◽  
G. B. Hartwig ◽  
R. A. Nielsen ◽  
A. P. Frost ◽  
A. D. Roses

Many genetic diseases can be demonstrated in skin cells cultured in vitro from patients with inborn errors of metabolism. Since myotonic muscular dystrophy (MMD) affects many organs other than muscle, it seems likely that this defect also might be expressed in fibroblasts. Detection of an alteration in cultured skin fibroblasts from patients would provide a valuable tool in the study of the disease as it would present a readily accessible and controllable system for examination. Furthermore, fibroblast expression would allow diagnosis of fetal and presumptomatic cases. An unusual staining pattern of MMD cultured skin fibroblasts as seen by light microscopy, namely, an increase in alcianophilia and metachromasia, has been reported; both these techniques suggest an altered glycosaminoglycan metabolism An altered growth pattern has also been described. One reference on cultured skin fibroblasts from a different dystrophy (Duchenne Muscular Dystrophy) reports increased cytoplasmic inclusions seen by electron microscopy. Also, ultrastructural alterations have been reported in muscle and thalamus biopsies from MMD patients, but no electron microscopical data is available on MMD cultured skin fibroblasts.


Author(s):  
J. R. Hully ◽  
K. R. Luehrsen ◽  
K. Aoyagi ◽  
C. Shoemaker ◽  
R. Abramson

The development of PCR technology has greatly accelerated medical research at the genetic and molecular levels. Until recently, the inherent sensitivity of this technique has been limited to isolated preparations of nucleic acids which lack or at best have limited morphological information. With the obvious exception of cell lines, traditional PCR or reverse transcription-PCR (RT-PCR) cannot identify the cellular source of the amplified product. In contrast, in situ hybridization (ISH) by definition, defines the anatomical location of a gene and/or it’s product. However, this technique lacks the sensitivity of PCR and cannot routinely detect less than 10 to 20 copies per cell. Consequently, the localization of rare transcripts, latent viral infections, foreign or altered genes cannot be identified by this technique. In situ PCR or in situ RT-PCR is a combination of the two techniques, exploiting the sensitivity of PCR and the anatomical definition provided by ISH. Since it’s initial description considerable advances have been made in the application of in situ PCR, improvements in protocols, and the development of hardware dedicated to in situ PCR using conventional microscope slides. Our understanding of the importance of viral latency or viral burden in regards to HIV, HPV, and KSHV infections has benefited from this technique, enabling detection of single viral copies in cells or tissue otherwise thought to be normal. Clearly, this technique will be useful tool in pathobiology especially carcinogenesis, gene therapy and manipulations, the study of rare gene transcripts, and forensics.


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