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Genetics and exercise: an introduction
Published in Adam P. Sharples, James P. Morton, Henning Wackerhage, Molecular Exercise Physiology, 2022
Claude Bouchard, Henning Wackerhage
CRISPR-Cas is commonly used to knockout or knock-in genes to test whether it affects a trait of interest in cells or organisms. CRISPR-Cas holds great promise for the treatment of Mendelian diseases such as Duchenne muscular dystrophy (55). On the downside, the comparatively easy usage of CRISPR-Cas can lead to misused and unethical applications. For instance, it could be used to create biological weapons, for personal biological enhancement or to modify genes for performance gain (i.e. gene doping, see below).
Should Genome Editing Replace Embryo Selection Following PGT?
Published in Carlos Simón, Carmen Rubio, Handbook of Genetic Diagnostic Technologies in Reproductive Medicine, 2022
In 2019, only seven years after CRISPR-Cas9-based genome editing was first described as a molecular biology tool (27,28), the birth of the first “CRISPR babies” Lulu and Nana was announced at a summit in Hong Kong. Gene editing had been applied at an embryonic stage in order to disrupt normal copies of the CCR5 gene, with the intention of conferring resistance to the HIV virus (the father of the children was HIV-positive). The announcement was met with condemnation from many quarters, not least from the scientific community. It was considered that the safety of the method had not been adequately demonstrated. In particular, there were concerns over the potential for inadvertent editing of unintended areas of the genome (i.e., off-target effects) and that there could be other unforeseen consequences of using CRISPR-Cas9 during early embryonic development. Additionally, many considered GE unnecessary, since there are well-validated “sperm washing” procedures, which avoid HIV transmission when using IVF. The two girls born as a result of the procedure are understood to have non-functional copies of CCR5. While this may provide the intended resistance to HIV infection, some evidence suggests there may also be negative health consequences of loss of CCR5 function, a problem that will be passed on to subsequent generations.
Healthy People / Immuno-enhancement
Published in Jonathan Anomaly, Creating Future People, 2020
The first genetically enhanced children were born in China, using techniques developed in the United States at Berkeley, Stanford, and MIT. In the Western world, strong laws and social norms prevent scientists from using CRISPR to edit human embryos intended for conception. While prohibitive laws also exist in China, Chinese scientists are under less scrutiny than Americans and Europeans.
Onasemnogene abeparvovec for the treatment of spinal muscular atrophy
Published in Expert Opinion on Biological Therapy, 2022
Hugh J. McMillan, Crystal M. Proud, Michelle A. Farrar, Ian E. Alexander, Francesco Muntoni, Laurent Servais
Currently, CRISPR technology is being investigated in several early stage clinical trials. Studies are investigating its use in the treatment of viral diseases, including human papillomavirus-related cervical neoplasia, refractory viral keratitis, human immunodeficiency virus, and coronavirus disease; solid tumors, including esophageal cancer, T- and B-cell malignancies, gastrointestinal malignancies, renal carcinoma, and tumors of the central nervous system; blood disorders, including leukemia and lymphoma, multiple myeloma, sickle cell disease, and β-thalassemia; and rare genetic disorders, including Kabuki Syndrome [137]. CRISPR technology has the opportunity to revolutionize treatment for countless patients and though its use in humans is limited, early results are promising.
Diagnostic accuracy of CRISPR technology for detecting SARS-CoV-2: a systematic review and meta-analysis
Published in Expert Review of Molecular Diagnostics, 2022
Xin Li, Huiling Zhang, Jing Zhang, Yang Song, Xuening Shi, Chao Zhao, Juan Wang
In 2013, Zhang et al. have firstly reported CRISPR-Cas Systems used in multiplex genome engineering, and later CRISPR technology came to apply for developing detection technology [8,9]. Diagnostic technology based on CRISPR-Cas can simultaneously satisfy a variety of detection criteria, which has the potential to be used to the next generation of diagnostic technology [10,11]. CRISPR Cas system can edit target DNA or RNA sequences with CRISPR Cas enzymology and amplification process in disease diagnosis platforms. Firstly, the vast amount of target nucleic acids synthesized with an amplification process, such as RT-RPA (reverse transcription recombinase polymerase amplification) or RT-LAMP [12]. Secondly, CRISPR RNA (crRNA) identifies the target nucleic acid for the spacer of the CRISPR Cas system and then the target nucleic acid was cut by specific Cas nucleases including Cas 9, Cas12 and Cas13 nucleases [12–14]. Finally, target nucleotides testing result was output by signal readout, such as fluorescence detection, lateral flow assay (LFA), and colorimetric aggregation with nanoparticles [12,15,16]. A large number of studies have reported that CRISPR technology has the advantages of low cost, ease to use, high sensitivity and specificity. To a certain extent, it can overcome the limitations of traditional molecular diagnostic methods such as RT-qPCR [17–19].
A toolmaker’s perspective on CRISPR-directed gene editing as a therapeutic strategy for leukemia and beyond
Published in Expert Review of Hematology, 2021
As exciting as all the remarkable advancements in the science of CRISPR have been, there is a reality to the application of gene editing in patients that is often overlooked. Many scientists would probably agree that it is too soon to move CRISPR-directed gene editing into clinical application, but there are hundreds of clinical protocols in process at the FDA, and several clinical trials at mid-phase [34]. At the present time, there are no reports of severe adverse effects when CRISPR is used as the main genetic tool, although more traditional gene therapy approaches continue to present problematic outcomes [35]. The advancement of gene editing into the clinic is no easy matter, and one of the most important hurdles has nothing to do with science but lives in the material world. Obtaining the proper licenses to produce a therapy that can be used in a designated patient population is complicated, expensive and, at best, confusing. The well-known CRISPR patent wars between Massachusetts Institute of Technology and the University of California at Berkeley have dominated the scientific legal docket. While this has certainly been entertaining to those of us who utilize this tool daily, the complex nature of licensing for use of this technology could prohibit important studies from going forward. Frankly, right now, it is not exactly clear how many licenses one would need to execute and produce a therapeutic product that can reach the people who need the treatment the most [36].