Emerging non-genetic therapies for sickle cell disease.

Emerging non-genetic therapies for sickle cell disease. Haematologica. 2019 Aug 14;: Authors: Carden MA, Little J Abstract Sickle cell disease afflicts millions of people worldwide and approximately 100,000 Americans. Complications are myriad and arise as a result of complex pathological pathways 'downstream' to a point mutation in DNA, and include red blood cell membrane damage, inflammation, chronic hemolytic anemia with episodic vaso-occlusion, ischemia and pain, and ultimately risk of cumulative organ damage with reduced patient lifespan. The National Heart, Lung, and Blood Institute's 2014 evidence-based guideline for sickle cell disease -management states additional research is needed before investigational curative therapies will be widely available to most patients with sickle cell disease. To date, sickle cell disease has been cured by hematopoietic stem cell transplantation in approximately 1000 people, most of whom were children, and significantly ameliorated by gene therapy in a handful of subjects who have only limited follow-up thus far. During a timespan in which over 20 agents were approved for Cystic Fibrosis by the Food and Drug Administration, similar approval was granted for only two drugs for sickle cell disease (hydroxyurea and L-glutamine) despite the higher prevalence of sickle cell disease. . This trajectory appears to be changing, as the lack of multimodal agent therapy in sickle cell disease has spurred engagement among many in...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research

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We report the first case of para-Bombay blood type in a Southeast Asian patient admitted at a tertiary hospital in Korea. A 23-year-old Indonesian man presented to the hospital with fever and was diagnosed with a disseminated nontuberculous mycobacterium infection and anemia. During blood group typing for blood transfusion, cell typing showed no agglutination with both anti-A and anti-B reagents. Serum typing showed strong reactivity against B cells and trace agglutination pattern with A1 cells. His red blood cells failed to react with anti-H reagents. Direct sequencing of FUT1 and FUT2 revealed a missense variation, c.328...
Source: Journal of Korean Medical Science - Category: Biomedical Science Tags: J Korean Med Sci Source Type: research
Contributors : Michael V Frochaux ; Maroun B Sleiman ; Bart DeplanckeSeries Type : Expression profiling by high throughput sequencingOrganism : Drosophila melanogasterResistance to enteric pathogens is a complex trait at the crossroads of multiple biological processes. We have previously shown in the Drosophila Genetic Reference Panel (DGRP) that resistance to infection is highly heritable, but our understanding of how the effects of genetic variants are channeled through distinct molecular layers is still limited. To address this, we employed a systems genetics approach on the gut transcriptomes from 38 control and orally...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Drosophila melanogaster Source Type: research
Authors: Horta-Baas G, Peláez-Ballestas I, Queipo G, Montero Hernández U, Romero-Figueroa MDS Abstract OBJECTIVES: Alexithymia is a personality trait related to the quality of life of women with fibromyalgia (FM). It is still unknown whether alexithymia is associated with the clinical manifestations of FM. The present study describes the relationship between alexithymia and the domains included in the core set recommended by the Outcome Measures in Rheumatology (OMERACT) for FM evaluation. METHODS: One hundred two women with FM were enrolled in the cross-sectional study. The domains evaluated were...
Source: Clinical and Experimental Rheumatology - Category: Rheumatology Tags: Clin Exp Rheumatol Source Type: research
Publication date: Available online 12 October 2019Source: Life SciencesAuthor(s): Liang Qiao, Yan Kong, Yingshen Shi, Aijun Sun, Ruijuan Ji, Chao Huang, Yuquan Li, Xiangqun YangAbstractAimsThe aims of the study are to investigate whether the combination of adipose-derived stem cells (ADSCs) with decellularized extracellular matrix (dECM) of myocardium can exert synergistic therapeutic effects on acute MI and the underlying mechanism.Main methodsMyocardial dECM from fresh porcine myocardium was prepared in an injectable gel, ADSCs were seeded into the myocardial dECM gels, and then the mixture was injected into the myocardi...
Source: Life Sciences - Category: Biology Source Type: research
Publication date: Available online 11 October 2019Source: Life SciencesAuthor(s): Mohammad Amin Javidi, Ayat Kaeidi, Seyedeh Sahar Mortazavi Farsani, Sadegh Babashah, Majid SadeghizadehAbstractAimsAn important obstacle on the way of cell-based therapy is the risk of tumorigenicity in the patients benefit from these transplanted cells due to undifferentiated cells which participate in transplantation. Curcumin, the main compound of spice turmeric -as one of the natural products-was demonstrated to possess effective anti-cancer properties, with no significant effect on normal cells in dose and/or time-dependent manner. Furth...
Source: Life Sciences - Category: Biology Source Type: research
ConclusionsPatients receiving perioperative gabapentin, acetaminophen, and NSAIDs under an ERAS protocol required significantly fewer narcotics and shorter length of stay. This protocol may merit consideration for use at other centers.
Source: The American Journal of Surgery - Category: Surgery Source Type: research
This report would examine developments at the state and federal-level, court cases, and current views from stakeholders. Policy Questions Which states have PAS laws and what do those laws provide? What protections against abuse of PAS?What have the Supreme Court and lower courts held regarding individuals’ rights under PAS laws? The laws themselves?Is there evidence that persons with disabilities are being denied treatment by insurance companies but offered PAS instead, as NCD predicted?How is PAS viewed by disability organizations? Has this evolved in the past 13 years? If so why? If not, why?Are persons with disabi...
Source: blog.bioethics.net - Category: Medical Ethics Authors: Tags: Health Care syndicated Source Type: blogs
Do genetic tests help in preparing for potential future health issues or open Pandora’s box full of concerns, worries and hypochondriac thoughts? Would you want to know your genetic fate? Whether you are at risk for Alzheimer’s or a chronic disease 30 years in advance? Would you want to live with this kind of information? Would you take the BRCA test to find out that you are at risk for breast cancer? What would you do if you were? The Medical Futurist team contemplated situations requiring hard, life-altering decisions. What would you do? Our genetic heritage carries secrets that are difficult to process In Se...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Bioethics Genomics alzheimer disease DNA dna testing doctor-patient doctor-patient relationship DTC future genetics Huntington's patient empowerment personal genomics Source Type: blogs
Follow the current debate on “healthcare reform”–which has NOTHING to do with healthcare reform, but healthcare insurance reform, by the way–and you will hear comments about the escalating and uncontrolled cost of healthcare and how people need access to it. What you will NOT hear is that fact that, because the healthcare system fails to deliver genuine health, real health is actually quite easy, straightforward, and inexpensive–nearly free. We achieve a life of being Undoctored, not becoming a profit source for the healthcare industry, not being subjected to the predatory practices of Big P...
Source: Wheat Belly Blog - Category: Cardiology Authors: Tags: Undoctored Wheat Belly Lifestyle acid reflux anti-aging autoimmune blood sugar bowel flora cholesterol Dr. Davis energy Gliadin gluten gluten-free grain-free grains health healthcare Inflammation joint pain low-carb Source Type: blogs
CONCLUSIONS: There is evidence to suggest that hydroxyurea is effective in decreasing the frequency of pain episodes and other acute complications in adults and children with sickle cell anaemia of HbSS or HbSβºthal genotypes and in preventing life-threatening neurological events in those with sickle cell anaemia at risk of primary stroke by maintaining transcranial doppler velocities. However, there is still insufficient evidence on the long-term benefits of hydroxyurea, particularly in preventing chronic complications of SCD, recommending a standard dose or dose escalation to maximum tolerated dose. There is al...
Source: Cochrane Database of Systematic Reviews - Category: Journals (General) Authors: Tags: Cochrane Database Syst Rev Source Type: research
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