CRISPR Gene Editing Is Being Tested in Human Patients, and the Results Could Revolutionize Health Care

It’s only been seven years since scientists first learned how to precisely and reliably splice the human genome using a tool called CRISPR, making it possible to think about snipping out disease-causing mutations and actually cure, once and for all, genetic diseases ranging from sickle cell anemia to certain types of cancer and even blindness. Doctors are plunging ahead in search of ways to use the relatively new technology to start treating patients. In China last November, scientist Jiankui He stunned—and dismayed—the genetic community when he announced he had already used CRISPR, which many believe still hasn’t been proven either safe or effective in human patients, to permanently alter the genomes of twin girls to be immune to HIV infection. He’s experiment was criticized because he edited the twins’ cells when they were embryos, therefore ensuring that every one of their cells is now changed, including their reproductive ones, which means their edited genomes can be passed on to their children, despite the fact that experts can’t be sure what the long term effects of such lasting modifications might be. Editas Medicine and Allergan recently announced a more acceptable form of gene editing, one that would change genetic defects in cells that don’t get passed onto the next generation. They are enrolling patients born with a congenital vision disease into what will be the first test in the U.S. of whether CRISPR can fix a mut...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized crispr Genetics Source Type: news

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Publication date: Available online 18 November 2019Source: Biochimica et Biophysica Acta (BBA) - Molecular and Cell Biology of LipidsAuthor(s): Zhen Wang, Hui Gyu Park, Dong Hao Wang, Riki Kitano, Kumar S.D. Kothapalli, J. Thomas BrennaAbstractBranched chain fatty acids (BCFA) and linear chain/normal odd chain fatty acids (n-OCFA) are major fatty acids in human skin lipids, especially sebaceous gland (SG) wax esters. Skin lipids contain variable amounts of monounsaturated BCFA and n-OCFA, in some reports exceeding over 20% of total fatty acids. Fatty acid desaturase 2 (FADS2) codes for a multifunctional enzyme that catalyz...
Source: Biochimica et Biophysica Acta (BBA) Molecular and Cell Biology of Lipids - Category: Lipidology Source Type: research
This study evaluated outcomes associated with treatment strategies for this nonagenarian population.MethodsTreatment and overall survival for patients 90 years and older with NSCLC in the National Cancer Data Base (2004-2014) were evaluated using logistic regression, the Kaplan-Meier method, and multivariable Cox proportional hazard models.ResultsThe majority (n = 4152, 57.6%) of the 7205 patients 90 years or older with stage I-IV NSCLC did not receive any therapy. For the entire cohort, receiving treatment was associated with significantly better survival when compared with no therapy (5-year survival, 9.3% [95% confidenc...
Source: The Annals of Thoracic Surgery - Category: Cardiovascular & Thoracic Surgery Source Type: research
ConclusionsThe delay in surgical resection of StageIIIa NSCLC is not associated increased early mortality, however it is associated with worse 3-year post-resection survival.
Source: The Annals of Thoracic Surgery - Category: Cardiovascular & Thoracic Surgery Source Type: research
Publication date: March 2020Source: Interdisciplinary Neurosurgery, Volume 19Author(s): Joel Kaye, Ahmed Meleis, Shabbar Danish, Zhenggang XiongAbstractMeningiomas are relatively slow-growing, typically benign tumors that arise from the arachnoid cells which form the meninges. They are the most common primary brain tumor and account for nearly one-third of all primary brain and spine tumors. Meningiomas can arise from any dural site, however they are infrequently found at the cerebellopontine angle (CPA). While WHO grade I meningiomas are benign and the slowest growing, WHO grade II meningiomas grow significantly faster, d...
Source: Interdisciplinary Neurosurgery - Category: Neurosurgery Source Type: research
s N Abstract Hypertension is the first modifiable vascular risk factor accounting for 10.4 million deaths worldwide; it is strongly and independently associated with the risk of stroke and is related to worse prognosis. In addition, hypertension seems to be a key player in the implementation of vascular cognitive impairment. Long-term hypertension, complicated or not by the occurrence of ischemic stroke, is often reviewed on its vascular side, and parenchymal consequences are put aside. Here, we sought to review the impact of isolated hypertension or hypertension associated to stroke on brain atrophy, neuron conne...
Source: Neural Plasticity - Category: Neurology Authors: Tags: Neural Plast Source Type: research
Abstract The transplantation of neural stem cells (NSCs) has become an emerging treatment for neural degeneration. A key factor in such treatments is to manipulate NSC behaviors such as proliferation and differentiation, resulting in the eventual regulation of NSC fate. Novel bionanomaterials have shown usefulness in guiding the proliferation and differentiation of NSCs due to the materials' unique morphological and topological properties. Among the nanomaterials, graphene has drawn increasing attention for neural regeneration applications based on the material's excellent physicochemical properties, surface modif...
Source: Neural Plasticity - Category: Neurology Authors: Tags: Neural Plast Source Type: research
Publication date: 20 September 2019Source: Journal of Molecular Biology, Volume 431, Issue 20Author(s): Ludovic SauguetAbstractDNA and RNA polymerases (DNAP and RNAP) play central roles in genome replication, maintenance and repair, as well as in the expression of genes through their transcription. Multisubunit RNAPs carry out transcription and are represented, without exception, in all cellular life forms as well as in nucleo-cytoplasmic DNA viruses. Since their discovery, multisubunit RNAPs have been the focus of intense structural and functional studies revealing that they all share a well-conserved active-site region c...
Source: Journal of Molecular Biology - Category: Molecular Biology Source Type: research
In conclusion, data presented in the current study suggest that tobacco smoking might cause alveolar tissue inflammation and vascular injury causing an immune response that elevates the white blood cells count. Another suggestion is that tobacco smoking defects the pulmonary gaseous exchange mechanism leading to the secondary polycythemia indicated by the increase in red blood cell count, hemoglobin levels, hematocrit and by the low serum erythropoietin levels.
Source: Saudi Journal of Biological Sciences - Category: Biology Source Type: research
We examined the efficacy of monosodium luminol-GVT (MSL), a drug that promotes redox homeostasis, for improving cognitive and mood function in GWI rats. Young rats were exposed to GWI-related chemicals and moderate restraint stress for four weeks. Four months later, GWI rats received different doses of MSL or vehicle for eight weeks. Behavioral analyses in the last three weeks of treatment revealed that GWI rats receiving higher doses of MSL displayed better cognitive and mood function associated with reinstatement of redox homeostasis. Such restoration was evident from the normalized expression of multiple genes encoding ...
Source: Redox Biology - Category: Biology Source Type: research
In this study, we report that the dietary isoflavone genistein (GN) synergistically improved antineoplasticity of CC in breast cancer by arresting cells at G2/M phase culminating in ROS dependent apoptosis. The combination of CC plus GN caused dysregulation of Bax and Bcl-2 ratio inducing mitochondrial dysfunction, activation of Caspase-3/7, -9 and PARP cleavage. Further, combination significantly suppresses phosphorylation of PI3K/Akt/NF-κB, enhancing apoptosis. Additionally, combination markedly reduced tumor growth compared to CC and GN alone in mouse 4T1 breast tumor model.SignificanceTogether, these studies sugg...
Source: Life Sciences - Category: Biology Source Type: research
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