Drugs for gene therapy: features of usage in geriatrics and pediatrics

2020 ◽  
Vol 18 (3) ◽  
pp. 237-244
Author(s):  
Konstantin G. Gurevich ◽  
Yulya A. Sorokina ◽  
Alexander L. Urakov ◽  
Darya M. Gavrilova ◽  
Lyubov V. Lovtsova ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Modern Medicine ◽  
Mechanisms Of Action ◽  
Gene Engineering ◽  
Pharmaceutical Substance ◽  
Genetic Sequence ◽  
Existing Problems ◽  
Therapeutic Drugs ◽  
Definition Of

Advances in modern medicine and biotechnology allow specialists to adjust the patients proteome and metabolome. Gene engineering allows us to create drugs that affect the cause of the disease at the level of gene expression. Thus, not the links of pathogenesis or the symptom of the disease are subjected to correction, but the trigger itself, a defective gene that provokes a cascade of pathological processes. According to the definition of the State Pharmacopoeia, gene therapeutic drugs are drugs whose pharmaceutical substance is a recombinant nucleic acid or includes a recombinant nucleic acid that allows for the regulation, repair, replacement, addition or removal of a genetic sequence. The article reflects all available, developed and used in real clinical practice gene therapy drugs of russian and foreign production. The mechanisms of action, features of the use of these drugs in pediatric and geriatric practice, as well as existing problems and limitations of their use, including deontological issues, are noted.

An RNA/DNA hybrid origami-based nanoplatform for efficient gene therapy

Nanoscale ◽  
2021 ◽  
Author(s):  
Xiaohui Wu ◽  
Qing Liu ◽  
Fengsong Liu ◽  
Tiantian Wu ◽  
Yingxu Shang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Homologous Gene ◽  
Target Mrna ◽  
Therapeutic Drugs ◽  
Efficient Gene

Nucleic acid nanostructures are promising biomaterials for the delivery of homologous gene therapeutic drugs. Herein, we report a facile strategy for the construction of target mRNA (scaffold) and antisenses (staple...

Nucleic acid-based antiviral and gene therapy of chronic hepatitis B infection

1997 ◽  
Vol 12 (9-10) ◽  
pp. S354-S369 ◽  
Author(s):  
JR WANDS ◽  
M GEISSLER ◽  
JZU PUTLITZ ◽  
H BLUM ◽  
F WEIZSÄCKER ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Chronic Hepatitis ◽  
Nucleic Acid ◽  
Hepatitis B ◽  
Chronic Hepatitis B ◽  
Hepatitis B Infection ◽  
Chronic Hepatitis B Infection

scholarly journals Activity and Mechanism of Action of Antifungal Peptides from Microorganisms: A Review

Molecules ◽  
2021 ◽  
Vol 26 (11) ◽  
pp. 3438
Author(s):  
Tianxi Li ◽  
Lulu Li ◽  
Fangyuan Du ◽  
Lei Sun ◽  
Jichao Shi ◽  
...  
Keyword(s):  
Mechanisms Of Action ◽  
Antifungal Drugs ◽  
Drug Resistant ◽  
Important Research ◽  
Factors Affecting ◽  
Natural Substances ◽  
Therapeutic Drugs ◽  
Antifungal Peptides ◽  
Control Work ◽  
And Control

Harmful fungi in nature not only cause diseases in plants, but also fungal infection and poisoning when people and animals eat food derived from crops contaminated with them. Unfortunately, such fungi are becoming increasingly more resistant to traditional synthetic antifungal drugs, which can make prevention and control work increasingly more difficult to achieve. This means they are potentially very harmful to human health and lifestyle. Antifungal peptides are natural substances produced by organisms to defend themselves against harmful fungi. As a result, they have become an important research object to help deal with harmful fungi and overcome their drug resistance. Moreover, they are expected to be developed into new therapeutic drugs against drug-resistant fungi in clinical application. This review focuses on antifungal peptides that have been isolated from bacteria, fungi, and other microorganisms to date. Their antifungal activity and factors affecting it are outlined in terms of their antibacterial spectra and effects. The toxic effects of the antifungal peptides and their common solutions are mentioned. The mechanisms of action of the antifungal peptides are described according to their action pathways. The work provides a useful reference for further clinical research and the development of safe antifungal drugs that have high efficiencies and broad application spectra.

Genetic manipulation on human beings

2010 ◽  
pp. 52-72
Author(s):  
Serena Zacchigna ◽  
Mauro Giacca
Keyword(s):  
Gene Therapy ◽  
Genetic Manipulation ◽  
Human Beings ◽  
Ethical Challenges ◽  
Genetic Characteristics ◽  
Safety Issues ◽  
The Public ◽  
Aesthetic Appearance ◽  
The Aesthetic ◽  
Definition Of

Since the early days of gene therapy, both the scientific community and the public have perceived the ethical challenges intrinsic to this discipline. First, the technology exploited by gene therapy is still experimental and burdened by important safety issues. Second, in several instances gene therapy aims at stably modifying the genetic characteristics of individuals. Third, the same modifications could in principle be applied also to embryos, foetuses or germ cells. Finally, while gene therapy applications are generally accepted for therapeutic purposes, the same gene transfer technologies could also be exploited to improve the aesthetic appearance, or the physical and intellectual performance of people. The definition of suitable guidelines for a controlled, ethically accepted translation of gene therapy to the clinics remains a major challenge for the near future.

GOAL SETTING AND ITS ACHIEVEMENT AS A CRITERION OF SCIENTIFIC RESEARCH IN THE FIELD OF CIVIL LAW

2021 ◽  
Vol 3 (3) ◽  
pp. 96-123
Author(s):  
L.V. Shchennikova
Keyword(s):  
Goal Setting ◽  
Social Relations ◽  
Civil Law ◽  
Scientific Research ◽  
Scientific Work ◽  
Point Of View ◽  
Existing Problems ◽  
The Creation ◽  
Scientific Results ◽  
Definition Of

Introduction: the article deals with the methodological problem of the meaning of the goal of civil law research. The author analyzes the dissertation abstracts from the point of view of goal setting, which were completed in different periods of the development of Russian civil law science, identifies the qualitative characteristics of the stages, and proves the connection of the achieved results with the researcher’s knowledge of the methodological methods of goal setting. Purpose: to show the value of goal setting in scientific research in general and in civil research in particular; to consider the relationship of goal setting with the achievement of specific scientific results on the examples of dissertations defended in the specialty 12.00.03; to justify the need to set as goals the fundamental problems associated with the identification of patterns of development of relations that are part of the subject of civil law regulation and the creation of effective mechanisms that mediate them. Methods: system-structural, system-functional, generalization, abstraction, analogy, logical, statistical, classification, legal modeling, comparative legal, forecasting, formal legal, historical. Results: civil methodology should take into account the importance of the goal in the organization of scientific work. Only a competent possession of goal setting skills can ultimately ensure the creation of scientifically-based mechanisms for effective impact of civil law norms on regulated social relations. Conclusions: 1) any science, including the science of civil law, is not only designed to study and describe existing problems, including legislative, doctrinal, and law enforcement. Research, in order to meet the criterion of scientific character, must attempt to identify the laws of development, both regulated relations and mechanisms that mediate them; 2) the significance of the goal in the development of science has been proven by outstanding philosophers. In addition, the very definition of science indicates that goal setting is one of its essential characteristics; 3) the analysis of the author’s abstracts of leading Russian tsivilists showed how the skilful setting of research goals helped to achieve them consistently, as well as to create a high-quality categorical apparatus of civil law science; 4) the analysis of modern dissertations showed that not all young researchers see the value of goal-setting and this methodological disadvantage is important for the author to eliminate.

scholarly journals Gene Therapy: A New Approach in Modern Medicine

2018 ◽  
Vol 5 (3) ◽  
pp. 106-117 ◽  
Author(s):  
Azam Yazdani ◽  
Zahra Alirezaie ◽  
Mohammad Javad Motamedi ◽  
Jafar Amani
Keyword(s):  
Gene Therapy ◽  
Modern Medicine ◽  
New Approach
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