scholarly journals Assembly PCR synthesis of optimally designed, compact, multi-responsive promoters suited to gene therapy application

2016 ◽  
Vol 6 (1) ◽  
Author(s):  
H. Mohamed ◽  
Y. Chernajovsky ◽  
D. Gould
Keyword(s):  
Gene Therapy ◽  
Gene Therapy Application ◽  
Assembly Pcr

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.

Gene Therapy: Paving New Roads in the Treatment of Hemophilia

2019 ◽  
Vol 45 (07) ◽  
pp. 743-750 ◽  
Author(s):  
Gabriela G. Yamaguti-Hayakawa ◽  
Margareth C. Ozelo
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Pediatric Patients ◽  
Viral Vector ◽  
Monogenic Disease ◽  
Adeno Associated Virus ◽  
Underlying Liver Disease ◽  
Experimental Animal Models ◽  
The Road ◽  
Gene Therapy Application

AbstractHemophilia is a monogenic disease with robust clinicolaboratory correlations of severity. These attributes coupled with the availability of experimental animal models have made it an attractive model for gene therapy. The road from animal models to human clinical studies has heralded significant successes, but major issues concerning a previous immunity against adeno-associated virus and transgene optimization remain to be fully resolved. Despite significant advances in gene therapy application, many questions remain pertaining to its use in specific populations such as those with factor inhibitors, those with underlying liver disease, and pediatric patients. Here, the authors provide an update on viral vector and transgene improvements, review the results of recently published gene therapy clinical trials for hemophilia, and discuss the main challenges facing investigators in the field.

Gene therapy for neurodegenerative and ocular diseases using lentiviral vectors

2005 ◽  
Vol 110 (1) ◽  
pp. 37-46 ◽  
Author(s):  
G. Scott Ralph ◽  
Katie Binley ◽  
Liang-Fong Wong ◽  
Mimoun Azzouz ◽  
Nicholas D. Mazarakis
Keyword(s):  
Gene Therapy ◽  
Nervous System ◽  
Viral Vectors ◽  
Viral Vector ◽  
Lentiviral Vectors ◽  
Great Promise ◽  
Ocular Diseases ◽  
Vector Systems ◽  
Gene Therapy Application ◽  
Wide Range

Gene therapy holds great promise for the treatment of a wide range of inherited and acquired disorders. The development of viral vector systems to mediate safe and long-lasting expression of therapeutic transgenes in specific target cell populations is continually advancing. Gene therapy for the nervous system is particularly challenging due to the post-mitotic nature of neuronal cells and the restricted accessibility of the brain itself. Viral vectors based on lentiviruses provide particularly attractive vehicles for delivery of therapeutic genes to treat neurological and ocular diseases, since they efficiently transduce non-dividing cells and mediate sustained transgene expression. Furthermore, novel routes of vector delivery to the nervous system have recently been elucidated and these have increased further the scope of lentiviruses for gene therapy application. Several studies have demonstrated convincing therapeutic efficacy of lentiviral-based gene therapies in animal models of severe neurological disorders and the push for progressing such vectors to the clinic is ongoing. This review describes the key features of lentiviral vectors that make them such useful tools for gene therapy to the nervous system and outlines the major breakthroughs in the potential use of such vectors for treating neurodegenerative and ocular diseases.

scholarly journals Healing the Fundamental Unit of Heredity (Gene Therapy): Current Perspective and What the Future Holds

2021 ◽  
Vol 5 (2) ◽  
pp. 62
Author(s):  
Bunga Anggreini Sari ◽  
Azalia Talitha Zahra ◽  
Ganda Purba Tasti ◽  
Ziske Maritska
Keyword(s):  
Gene Therapy ◽  
Cancer Cells ◽  
Genetic Recombination ◽  
Ex Vivo ◽  
Genetic Diseases ◽  
Fundamental Unit ◽  
Embryonic Cells ◽  
Gene Therapy Application ◽  
Wide Range ◽  
The Future

The ability to make precise adjustments to the human genome has been a goal of healing in which gene also introduces as the fundamental unit of heredity, in biomolecular technology in genetic diseases have opened new knowledge such as gene therapy. Gene therapy is a technique to repair DNA where its usage is to treat the malignancy and inherited genetic diseases. Gene therapy is a choice to the genetic cloth that goals to remedy a sickness this is hard to deal with or perhaps has no treatment. Currently, gene remedy is done in approaches to patients, specifically embryonic cells and somatic cells, every in vivo and ex vivo. Moral considerations with modification of the difficulty's cells and oversight of regulation and reagents want to be taken into consideration within the gene therapy project. Applications for using gene remedies have begun to be widely used, which include in case of maximum cancers, coronary heart disorder, infectious sicknesses, and others. Gene therapy has spread to a wide range of applications then go beyond the modification of genetic disorders. Advances in genetic modification of cancer cells and immunity and the use of viruses and bacteria to control cancer cells have resulted in many clinical trials and product developments for cancer treatment. The miracles and blessings of gene therapy are might believe, but even though they are being studied and developed now and, in the future, so that the desire for gene therapy may be even better future.Keywords: gene therapy, genetic recombination, gene therapy application

scholarly journals Retracted: Tumor Tropism of Intravenously Injected Human-Induced Pluripotent Stem Cell-Derived Neural Stem Cells and Their Gene Therapy Application in a Metastatic Breast Cancer Model

Stem Cells ◽  
2012 ◽  
Vol 30 (5) ◽  
pp. 1021-1029 ◽  
Author(s):  
Jing Yang ◽  
Dang Hoang Lam ◽  
Sally Sallee Goh ◽  
Esther Xingwei Lee ◽  
Ying Zhao ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Gene Therapy ◽  
Pluripotent Stem Cell ◽  
Metastatic Breast ◽  
Induced Pluripotent Stem Cell ◽  
Cancer Model ◽  
Gene Therapy Application ◽  
Breast Cancer Model ◽  
Tumor Tropism ◽  
Induced Pluripotent

Study of the construction of an adenoviral vector for hepatic tumor gene therapy application

2011 ◽  
Vol 44 (13) ◽  
pp. S204
Author(s):  
Bahare Mehrdad Vahdati ◽  
Hossein A. Tehrani ◽  
Seyed Younes Hosseini
Keyword(s):  
Gene Therapy ◽  
Adenoviral Vector ◽  
Hepatic Tumor ◽  
Tumor Gene Therapy ◽  
Gene Therapy Application ◽  
Tumor Gene
Sign in / Sign up

Export Citation Format

Share Document