Applications of genome editing technology in animal disease modeling and gene therapy
Publication date: Available online 8 June 2019Source: Computational and Structural Biotechnology JournalAuthor(s): Qian Li, Zhou Qin, Qingnan Wang, Ting Xu, Yang Yang, Zhiyao HeAbstractGenome editing technology is a technique for targeted genetic modifications, enabling the knockout and addition of specific DNA fragments. This technology has been widely used in various types of biomedical research, clinics and agriculture. In terms of disease research, constructing appropriate animal models is necessary. Combining reproductive technology with genome editing, many animal disease models have been generated for basic and clinical research. In addition, precisely targeted modifications allow genome editing to flourish in the field of gene therapy. Many mutations refractory to traditional gene therapy could be permanently corrected at the DNA level. Thus, genome editing is undoubtedly a promising technology for gene therapy. In this review, we mainly introduce the applications of genome editing in constructing animal disease models and gene therapies, as well as its future prospects and challenges.
Publication date: December 2019Source: Materials Science and Engineering: C, Volume 105Author(s): Rebeca T. Steipel, Matthew D. Gallovic, Cole J. Batty, Eric M. Bachelder, Kristy M. AinslieAbstractAlso known as electrospray, electrohydrodynamic atomization has been used extensively in the last 15 years to develop polymer-based particles for drug delivery in cell and animal models. More recently, novel core-shell, multi-axial, and other electrospray particles have been developed from an array of polymers for a variety of biomedical applications. This review focuses on electrospray as a novel method of particle fabrication...
Massive intraosseous vascular malformations, a relatively rare entity in the vascular malformation spectrum, deserves attention as involving the membranous bones of the craniofacial skeleton and may lead to severe life-threatening hemorrhages and even death. The main aim of this study was to summarize the 25 years of clinical experience with these vascular malformation osseous patients, focus on the molecular and genetic aspect of the clinical entity, and to emphasize the certain challenging conditions in the treatment of these patients. All the patients appeared to be unaffected at birth, whereas initial symptoms occasion...
Authors: Tse J, Singla N, Ghandour R, Lotan Y, Margulis V Abstract Introduction: The current first line therapy for high grade (HG) non-muscle invasive bladder cancer (NMIBC) is intravesical Bacillus Calmette-Guerin (BCG). Patients who recur or progress despite BCG are recommended to undergo radical cystectomy or participate in clinical trials. There is an urgent need for alternative therapies in the BCG-unresponsive NMIBC realm. Areas covered: We queried clinicaltrials.gov and pubmed.gov for current and recently completed early clinical trials pertaining to investigational agents used for the treatment of BCG-unre...
"If the products that the (FDA) is approving aren't standing the test of time, are they going to become more tentative with drug approvals? (Cell and gene therapies) may bring that issue to the forefront," one regulatory expert said.
CONCLUSIONS In hyperoxia-induced rat models of BPD, hBD2 promotes alveolarization and improves pulmonary function. The mechanism may contribute in alleviating inflammation response and inhibiting pro-inflammatory factors including IL-1ß, IL-6, and TNF-alpha. PMID: 31411185 [PubMed - in process]
Human Gene Therapy,Volume 30, Issue 8, Page 919-920, August 2019.
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Human Gene Therapy, Ahead of Print.