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...

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.

Efficient Gene Therapy of Pancreatic Cancer via a Peptide Nucleic Acid (PNA)‐Loaded Layered Double Hydroxides (LDH) Nanoplatform

Small ◽  
2020 ◽  
Vol 16 (23) ◽  
pp. 1907233 ◽  
Author(s):  
Zhiguo Yu ◽  
Ping Hu ◽  
Yingying Xu ◽  
Qunqun Bao ◽  
Dalong Ni ◽  
...  
Keyword(s):  
Pancreatic Cancer ◽  
Gene Therapy ◽  
Nucleic Acid ◽  
Layered Double Hydroxides ◽  
Peptide Nucleic Acid ◽  
Efficient Gene ◽  
Double Hydroxides

Genetic Engineering of AAV Capsid Gene for Gene Therapy Application

2020 ◽  
Vol 20 (5) ◽  
pp. 321-332
Author(s):  
Yunbo Liu ◽  
Xu Zhang ◽  
Lin Yang
Keyword(s):  
Gene Therapy ◽  
Neutralizing Antibodies ◽  
Human Subjects ◽  
Genetic Diseases ◽  
Capsid Gene ◽  
Human Populations ◽  
Stable Gene ◽  
Efficient Gene ◽  
Gene Therapy Application

Adeno-associated virus (AAV) is a promising vector for in vivo gene therapy because of its excellent safety profile and ability to mediate stable gene expression in human subjects. However, there are still numerous challenges that need to be resolved before this gene delivery vehicle is used in clinical applications, such as the inability of AAV to effectively target specific tissues, preexisting neutralizing antibodies in human populations, and a limited AAV packaging capacity. Over the past two decades, much genetic modification work has been performed with the AAV capsid gene, resulting in a large number of variants with modified characteristics, rendering AAV a versatile vector for more efficient gene therapy applications for different genetic diseases.

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

Efficient gene therapy based targeting system for the treatment of inoperable tumors

2012 ◽  
Vol 14 (4) ◽  
pp. 221-230 ◽  
Author(s):  
Thomas Wirth ◽  
Jere Tuomas Pikkarainen ◽  
Haritha Dhammika Samaranayake ◽  
Pauliina Lehtolainen-Dalkilic ◽  
Hanna Pirita Lesch ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Efficient Gene

AAV9 is considered the most efficient AAV serotype targeting blood-brain barriers. To enhance effective gene therapy for CNS illnesses, testing novel vectors with more efficient crossing capabilities is vital

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Intravenous Injection ◽  
Peripheral Circulation ◽  
Full Potential ◽  
Cns Diseases ◽  
Aav Vector ◽  
Early Clinical Trial ◽  
Blood Brain ◽  
Efficient Gene ◽  
Trial Outcomes

Although gene therapy for CNS diseases shows promise in cell and animal investigations, most human trials have failed to satisfy the requisite requirements. Finding novel techniques to boost the efficacy of gene therapy in treating CNS diseases is still crucial. A growing number of clinical trials have proved the efficacy and safety of using AAV vectors, making AAV vector research a gene therapy hotspot. However, due to the presence of the BBB, many siRNA and DNA with potential therapeutic value are difficult to transport from peripheral circulation to the brain using AAV vectors, limiting the clinical impact of gene therapy drugs in the CNS and posing a major challenge to the field of CNS gene therapy. In early studies, AAV9 was considered the most effective AAV serotype for getting through the blood-brain barrier and transduction to central nervous system cells following intravenous injection. Aavrh10 isolated from rhesus monkeys was equal to, if not superior to, AAV9. AAV-PHP.B, a newly built capsid, exhibits 40-fold greater efficacy than AAV9 in astrocyte and neuron transduction. AAV-PHP.eB, a modified AAV-PHP.B variety, was identified to retain PHP.B's AAV-capacity to transduce astrocytes while enhancing neuronal transduction. While the four serotypes AAV9, AAVrh10, AAV-PHP.B, and AAV-PHP.eB have been validated to penetrate mice's BBB following intravenous injection, the number of AAV vectors that can do so is low. Moreover, the manner in which AAV vectors penetrate the BBB remains unclear. To promote efficient gene therapy for CNS diseases, it is still important to test new vectors with more efficient crossing abilities and understand their crossing processes. In addition to technical challenges, AAV vectors in treating CNS diseases may be limited by cautious attitudes to innovative treatments. Continued advances in AAV vector research, together with early clinical trial outcomes, might help researchers achieve the full potential of AAV-based CNS disease therapies.

scholarly journals Sterile Abscess in the Myocardium after Direct Intramyocardial Injection Related to Gene Therapy in a Swine Model

2011 ◽  
Vol 2011 ◽  
pp. 1-2 ◽  
Author(s):  
Kiyotake Ishikawa ◽  
Dennis Ladage ◽  
Lisa Tilemann ◽  
Yoshiaki Kawase ◽  
Roger J. Hajjar
Keyword(s):  
Gene Therapy ◽  
Gene Transfer ◽  
Viral Vectors ◽  
Swine Model ◽  
Clinical Settings ◽  
Intramyocardial Injection ◽  
New Therapy ◽  
Sterile Abscess ◽  
Efficient Gene ◽  
Cardiac Gene

Cardiac gene therapy is one of the most promising approaches to cure patients with cardiac dysfunctions. Many ways of efficient gene transfer using viral vectors are tested, and some of them are already used in clinical settings. However, it is always important to be keenly alert to the possible complications when a new therapy is introduced. We present a case of myocardial sterile abscess in a swine model associated with a direct myocardial injection.

scholarly journals Design of time-delayed safety switches for CRISPR gene therapy

2021 ◽  
Author(s):  
Dashan Sun
Keyword(s):  
Gene Therapy ◽  
Immune Response ◽  
Time Delay ◽  
Cell Lines ◽  
Gene Editing ◽  
Drug Accumulation ◽  
Work Time ◽  
Crispr System ◽  
Efficient Gene ◽  
Target Effect

CRISPR system is a powerful gene editing tool which has already been reported to address a variety of gene relevant diseases in different cell lines. However, off-target effect and immune response caused by Cas9 remain two fundamental problems. In our work, time-delayed safety switches are designed based on either artificial ultrasensitivity transmission module or intrinsic time delay in biomolecular activities. By addressing gene therapy efficiency, off-target effect, immune response and drug accumulation, we hope our safety switches may offer inspiration in realizing safe and efficient gene therapy in humans.

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