Supersize My Mouse

2020 ◽  
pp. 31-57
Author(s):  
John Parrington

Although human beings have been altering genomes by selection and breeding of particular animal or plant variants for thousands of years, and X-rays and chemicals were first used to create mutants in the early 20th century genetic engineering in the true sense only became possible much more recently. First, the discovery that genes are made of DNA, revealed the material nature of the genome. Second, scientists in the early 1970s discovered enzymes in bacteria that can be used to cut and paste DNA in the test tube. Using such molecular ‘tools‘ bacteria were engineered to produce important medical products such as human insulin for diabetics, and from this was born what would become a billion-dollar biotechnology industry. A further important development was the discovery of embryonic stem cells and manipulation of these to make ‘knockout’ mice that had a deletion of a specific gene, or ‘knockin’ mice that had subtle changes in a gene. However such an approach was still relatively expensive and time-consuming, and cold only be applied to mice. And although gene constructs could be introduced into cells in a less precise manner, the crude nature of this approach limited its application for both agriculture and gene therapy. In both areas of application there have been concerns about the safety and ethics of using such an approach. A major criticism has been the lack of precision in where a gene construct would end up the genome, leading to concerns about possible adverse effects.

2021 ◽  
Vol 21 (4) ◽  
pp. 215-219
Author(s):  
A.K. Drakon ◽  
◽  
A.G. Kurguzova ◽  
V.M. Sheludchenko ◽  
N.B. Korchazhkina ◽  
...  

Age-related macular degeneration (AMD) is the leading cause of blindness in people over 55 in developed countries. Moreover, the number of these patients will increase growth as life expectancy increases. It is estimated that late AMD accounts for half of blindness and low vision cases in European countries. A myriad of studies is currently underway to discover cutting-edge, effective therapeutic modalities. Gene therapy is a novel alternative to regular intravitreal injections of anti-VEGF agents for late wet AMD. This technique’s heart is a specific gene delivery to target cells to generate natural VEGF inhibitors. Gene therapy affecting the complement system to deactivate its end product, the membrane attack complex, is reasonable in late atrophic AMD. Studies on stem cell therapy for late atrophic AMD undergo as well. It was demonstrated that retinal pigment epithelium (RPE) cells derived from human embryonic stem cells or induced pluripotent stem cells express typical RPE markers that can phagocytize photoreceptor segments. Electrical stimulation and magnet therapy are already introduced into clinical practice to rehabilitate patients with late AMD. Magnetic and electrical fields improve impulse transmitting, activate intracellular and tissue regeneration of the retina. Recent findings are promising but require further in-depth studies. Keywords: age-related macular degeneration, retinal scar, gene therapy, stem cells, physiotherapy, rehabilitative medicine. For citation: Drakon A.K., Kurguzova A.G., Sheludchenko V.M., Korchazhkina N.B. Non-medical treatment for late age-related macular degeneration. Russian Journal of Clinical Ophthalmology. 2021;21(4):215–219 (in Russ.). DOI: 10.32364/2311-7729-2021-21-4-215-219.


2021 ◽  
Vol 12 (1) ◽  
Author(s):  
Xia Wang ◽  
Jin Wang ◽  
Yu-Man Tsui ◽  
Chaoran Shi ◽  
Ying Wang ◽  
...  

AbstractGrowing evidences suggest that cancer stem cells exhibit many molecular characteristics and phenotypes similar to their ancestral progenitor cells. In the present study, human embryonic stem cells are induced to differentiate into hepatocytes along hepatic lineages to mimic liver development in vitro. A liver progenitor specific gene, RALY RNA binding protein like (RALYL), is identified. RALYL expression is associated with poor prognosis, poor differentiation, and metastasis in clinical HCC patients. Functional studies reveal that RALYL could promote HCC tumorigenicity, self-renewal, chemoresistance, and metastasis. Moreover, molecular mechanism studies show that RALYL could upregulate TGF-β2 mRNA stability by decreasing N6-methyladenosine (m6A) modification. TGF-β signaling and the subsequent PI3K/AKT and STAT3 pathways, upregulated by RALYL, contribute to the enhancement of HCC stemness. Collectively, RALYL is a liver progenitor specific gene and regulates HCC stemness by sustaining TGF-β2 mRNA stability. These findings may inspire precise therapeutic strategies for HCC.


2011 ◽  
Vol 22 (11) ◽  
pp. 1365-1377 ◽  
Author(s):  
Xiao Ying Bak ◽  
Hoang Lam Dang ◽  
Jingye Yang ◽  
Kai Ye ◽  
Esther X.W. Lee ◽  
...  

2006 ◽  
Vol 10 (15) ◽  
pp. 818-833

Aida Pharmaceuticals Announces Completion of Phase I Trials For Gene-Therapy Drug. Neusoft Inks JV with Trivitron, to invest US $5 Million for Medical Equipment Unit in India. Makoto Signs $20 Million Research Collaboration with Taiho Pharmaceutical Co Ltd. Fukuda Denshi to Sell Kontron Medical SAS and Kontron Medical AG. ESI First in the World to Create Embryonic Stem Cells for Clinical Use. Latest Innovation Enables Early Diagnosis of Dengue. Exploit Technologies Presents Flagship Programs & Incubation Projects at Inaugural Venture Capital Forum. Merck Sharp & Dohme Expands Singapore Facility with S$100 Million Investment. MedMira—Innovation Inspired by People.


2020 ◽  
Vol 15 (4) ◽  
pp. 379-387
Author(s):  
Aryendu K. Saini ◽  
Rakesh Saini ◽  
Himanshu Bansode ◽  
Anurag Singh ◽  
Lalita Singh

Stem cells are the undifferentiated cells in the body that possess the ability to differentiate and give rise to any type of cells in the body. In recent years, there has been a growing interest in therapies involving stem cells as different treatment methods got developed. Depending on the source, there are two major kinds of stem cells, embryonic and adult stem cells. The former type is found in the embryo at the different developmental stages before the implantation and excels the latter owing to pluripotency. On the premise of the attributes of stem cells, they are touted as the "panacea for all ills" and are extensively sought for their potential therapeutic roles. There are a lot of robust pieces of evidence that have proved to cure the different ailments in the body like Huntington disease, Parkinson's disease, and Spinal cord injury with stem cell therapy but associated with adverse effects like immune rejection and teratoma formation. In this regard, the pre-morula (isolated at an early pre-morula stage) stem cells (PMSCs) are one of its kind of embryonic stem cells that are devoid of the aforementioned adverse effects. Taking the beneficial factor into account, they are being used for the treatment of disorders like Cerebral palsy, Parkinson's disorder, Aplastic anemia, Multiple sclerosis and many more. However, it is still illegal to use stem cells in the abovementioned disorders. This review encompasses different stem cells and emphasizes on PMSCs for their uniqueness in therapy as no other previously published literature reviews have taken these into consideration. Later in the review, current regulatory aspects related to stem cells are also considered.


Sign in / Sign up

Export Citation Format

Share Document