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Genetic therapy

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https://www.readbyqxmd.com/read/29792808/eradication-of-established-tumors-by-chemically-self-assembled-nanoring-csan-targeted-t-cells
#1
Jacob Petersburg, Jingjing Shen, Clifford M Csizmar, Katherine A Murphy, Justin Spanier, Kari Gabrielse, Thomas S Griffith, Brian Fife, Carston R Wagner
Our laboratory has developed chemically self-assembled nanorings (CSANs) as prosthetic antigen receptors (PARs) for the non-genetic modification of T-cell surfaces. PARs have been successfully employed in vitro to activate T cells for the selective killing of leukemia cells. However, PAR efficacy has yet to be evaluated in vivo or against solid tumors. Therefore, we developed bispecific PARs that selectively target the human CD3 receptor and human Epithelial Cell Adhesion Molecule (EpCAM), which is overexpressed on multiple carcinomas and cancer stem cells...
May 24, 2018: ACS Nano
https://www.readbyqxmd.com/read/29792527/enteric-neural-stem-cells-expressing-insulin-like-growth-factor-1-a-novel-cellular-therapy-for-hirschsprung-s-disease-in-mouse-model
#2
Wei Liu, Lijuan Zhang, Rongde Wu
Transplanting enteric neural stem cells (ENSCs) is an innovative approach for replacing enteric neurons in Hirschsprung's disease (HSCR). However, posttransplantation cell survival and differentiation limit efficacy. We aimed to investigate whether transplantation of ENSCs engineered with insulin-like growth factor 1 (IGF-1) could improve survival and differentiation of the engrafted cells and promote functional recovery of the aganglionic colon. ENSCs were isolated from the intestine of neonatal mice and genetically modified to express IGF-1...
May 24, 2018: DNA and Cell Biology
https://www.readbyqxmd.com/read/29791948/multiple-sclerosis
#3
Bassem I Yamout, Raed Alroughani
Multiple sclerosis (MS) is a chronic central nervous system inflammatory disease of autoimmune etiology, mediated by activated T cells with evolving evidence of a significant contribution from B cells and cells of the innate immune system. The disease is thought to be due to a complex interaction between different genetic and environmental factors. The prevalence of MS is rising all over the world, due on one hand to earlier diagnosis and prolonged survival, and on the other to a true increase in incidence of the disease...
April 2018: Seminars in Neurology
https://www.readbyqxmd.com/read/29791608/hiv-1-genetic-diversity-and-resistance-to-antiretroviral-drugs-among-pregnant-women-in-ribeir%C3%A3-o-preto-sp-brazil-cross-sectional-study
#4
Ana Teresa Mancini Pimenta, Isadora Alonso Correa, Patricia Pereira Dos Santos Melli, Renata Abduch, Geraldo Duarte, José Carlos Couto-Fernandez, Silvana Maria Quintana
BACKGROUND: Increasing genetic diversity of HIV-1 and emergence of drug-resistant mutations may reduce the efficacy of antiretroviral therapy and prophylaxis that are used to prevent mother-to-child transmission. The aim of this study was to assess the genetic diversity and prevalence of drug-resistant mutations among HIV-infected pregnant women. DESIGN AND SETTING: Cross-sectional study at an outpatient clinic for infectious diseases within gynecology and obstetrics...
March 2018: São Paulo Medical Journal, Revista Paulista de Medicina
https://www.readbyqxmd.com/read/29791243/amcp-partnership-forum-managing-care-in-the-wave-of-precision-medicine
#5
(no author information available yet)
Precision medicine, the customization of health care to an individual's genetic profile while accounting for biomarkers and lifestyle, has increasingly been adopted by health care stakeholders to guide the development of treatment options, improve treatment decision making, provide more patient-centered care, and better inform coverage and reimbursement decisions. Despite these benefits, key challenges prevent its broader use and adoption. On December 7-8, 2017, the Academy of Managed Care Pharmacy convened a group of stakeholders to discuss these challenges and provide recommendations to facilitate broader adoption and use of precision medicine across health care settings...
May 23, 2018: Journal of Managed Care & Specialty Pharmacy
https://www.readbyqxmd.com/read/29790918/temporal-and-tissue-specific-variability-of-smn-protein-levels-in-mouse-models-of-spinal-muscular-atrophy
#6
Ewout Jn Groen, Elena Perenthaler, Natalie L Courtney, Crispin Y Jordan, Hannah K Shorrock, Dinja van der Hoorn, Yu-Ting Huang, Lyndsay M Murray, Gabriella Viero, Thomas H Gillingwater
Spinal muscular atrophy (SMA) is a progressive motor neuron disease caused by deleterious variants in SMN1 that lead to a marked decrease in survival motor neuron (SMN) protein expression. Humans have a second SMN gene (SMN2) that is almost identical to SMN1. However, due to alternative splicing the majority of SMN2 mRNA is translated into a truncated, unstable protein that is quickly degraded. Because the presence of SMN2 provides a unique opportunity for therapy development in SMA patients, the mechanisms that regulate SMN2 splicing and mRNA expression have been elucidated in great detail...
May 22, 2018: Human Molecular Genetics
https://www.readbyqxmd.com/read/29790419/rictor-gene-amplification-is-correlated-with-metastasis-and-therapeutic-resistance-in-triple-negative-breast-cancer
#7
Said El Shamieh, Fatima Saleh, Salim Moussa, Joseph Kattan, Fadi Farhat
Triple-negative breast cancer (TNBC) is characterized by its aggressive behavior, metastasis and lack of targeted therapies. Herein, we discuss the clinical, histopathological and genetic profile of a woman diagnosed with TNBC. Since the patient had no durable response to chemotherapy, a genetic profiling was carried out. Next-generation sequencing analysis of 592 genes showed a missense mutation, p.E545A in PIK3CA, thus the patient was started on the mTOR inhibitor everolimus, in combination with exemestane, which controlled her pain; however, the disease progressed aggressively...
May 23, 2018: Pharmacogenomics
https://www.readbyqxmd.com/read/29790402/vitamin-d-pathway-gene-polymorphisms-affecting-daclatasvir-plasma-concentration-at-2-weeks-and-1-month-of-therapy
#8
Jessica Cusato, Amedeo De Nicolò, Lucio Boglione, Fabio Favata, Alessandra Ariaudo, Simone Mornese Pinna, Chiara Carcieri, Federica Guido, Giuseppe Cariti, Giovanni Di Perri, Antonio D'Avolio
AIM: Vitamin D (VD) influences genetic expression through its receptor (VDR). VD pathway gene polymorphisms seem to influence antiviral drug pharmacokinetics and therapeutic outcome/toxicity. We investigated the association between daclatasvir (DCV) plasma concentrations and genetic variants (SNPs) associated with the VD pathway. PATIENTS & METHODS: Chronic hepatitis C patients treated with DCV from 2014 to 2016 were included. Genotypes were assessed through real-time PCR and plasma concentrations through liquid chromatography...
June 1, 2018: Pharmacogenomics
https://www.readbyqxmd.com/read/29790357/-control-editing-of-the-genome-within-reach-or-already-in-our-hands
#9
Radim Brdička, Radoslav Omelka
Although different genome editing tools have been around for decades, the recent emergence of cheap, quick, and accessible CRISPR/Cas9 technology has led to a revolution in this field. The technique has the potential to transform medicine from curative into preventive using a gene therapy. An application of genome editing has proven to be effective for both genetic and non-genetic (e.g. infectious) diseases. However, cancer and rare diseases treatment is at the forefront of interest. Concurrently, the legal and ethical frameworks should be discussed, especially as the technology moves towards a modification of the germ cells or embryos...
2018: Casopís Lékar̆ů C̆eských
https://www.readbyqxmd.com/read/29790354/-opioids-and-new-trends-in-pain-therapy
#10
Jan Lejčko
Opioids are well known for their ability to reduce the perception of pain without a loss of consciousness. However, the influence of opioids on organism is very complex. Clinical pharmacology of currently available opioid analgesics is determined by three major factors - their opioid receptor properties, pharmacokinetic properties and genetic polymorphism in the opiate receptor and cytochrome P450 isoform. In acute severe pain are opioids very efficient. Long-term experience with opioid analgesia in patients with cancer pain has shown high favorable risk/benefit ratio...
2018: Casopís Lékar̆ů C̆eských
https://www.readbyqxmd.com/read/29790107/the-role-of-pharmacogenomics-in-bipolar-disorder-moving-towards-precision-medicine
#11
REVIEW
Claudia Pisanu, Urs Heilbronner, Alessio Squassina
Bipolar disorder (BD) is a common and disabling psychiatric condition with a severe socioeconomic impact. BD is treated with mood stabilizers, among which lithium represents the first-line treatment. Lithium alone or in combination is effective in 60% of chronically treated patients, but response remains heterogenous and a large number of patients require a change in therapy after several weeks or months. Many studies have so far tried to identify molecular and genetic markers that could help us to predict response to mood stabilizers or the risk for adverse drug reactions...
May 22, 2018: Molecular Diagnosis & Therapy
https://www.readbyqxmd.com/read/29789421/pericytes-in-the-premetastatic-niche
#12
REVIEW
Ana E Paiva, Luiza Lousado, Daniel A P Guerra, Patrick O Azevedo, Isadora F G Sena, Julia P Andreotti, Gabryella S P Santos, Ricardo Gonçalves, Akiva Mintz, Alexander Birbrair
The premetastatic niche formed by primary tumor-derived molecules contributes to fixation of cancer metastasis. The design of efficient therapies is limited by the current lack of knowledge about the details of cellular and molecular mechanisms involved in the premetastatic niche formation. Recently, the role of pericytes in the premetastatic niche formation and lung metastatic tropism was explored by using state-of-the-art techniques, including in vivo lineage-tracing and mice with pericyte-specific KLF4 deletion...
May 22, 2018: Cancer Research
https://www.readbyqxmd.com/read/29789416/macrophage-derived-granulin-drives-resistance-to-immune-checkpoint-inhibition-in-metastatic-pancreatic-cancer
#13
Michael C Schmid, Valeria Quaranta, Carolyn Rainer, Sebastian Rune Nielsen, Meirion Llŷr Raymant, Muhammad S Ahmed, Dannielle D Engle, Arthur Taylor, Trish Murray, Fiona Campbell, Daniel H Palmer, David A Tuveson, Ainhoa Mielgo
The ability of disseminated cancer cells to evade the immune response is a critical step for efficient metastatic progression. Protection against an immune attack is often provided by the tumor microenvironment that suppresses and excludes cytotoxic CD8+ T cells. Pancreatic ductal adenocarcinoma (PDAC) is a highly aggressive metastatic disease with unmet needs, yet the immunoprotective role of the metastatic tumor microenvironment in pancreatic cancer is not completely understood. In this study we find that macrophage-derived granulin contributes to cytotoxic CD8+ T cell exclusion in metastatic livers...
May 22, 2018: Cancer Research
https://www.readbyqxmd.com/read/29788888/scap-srebps-are-central-players-in-lipid-metabolism-and-novel-metabolic-targets-in-cancer-therapy
#14
Xiang Cheng, Jianying Li, Deliang Guo
Lipid metabolism reprogramming emerges as a new hallmark of malignancies. Sterol regulatory element-binding proteins (SREBPs), which are central players in lipid metabolism, are endoplasmic reticulum (ER)-bound transcription factors that control the expression of genes important for lipid synthesis and uptake. Their transcriptional activation requires binding to SREBP cleavage-activating protein (SCAP) to translocate their inactive precursors from the ER to the Golgi to undergo cleavage and subsequent nucleus translocation of their NH2-terminal forms...
May 22, 2018: Current Topics in Medicinal Chemistry
https://www.readbyqxmd.com/read/29788244/nonsynonymous-polymorphism-in-guanine-monophosphate-synthetase-is-a-risk-factor-for-unfavorable-thiopurine-metabolite-ratios-in-patients-with-inflammatory-bowel-disease
#15
Rebecca L Roberts, Mary C Wallace, Margien L Seinen, Adriaan A van Bodegraven, Krupa Krishnaprasad, Gregory T Jones, Andre M van Rij, Angela Baird, Ian C Lawrance, Ruth Prosser, Peter Bampton, Rachel Grafton, Lisa A Simms, Corrie Studd, Sally J Bell, Martin A Kennedy, Jacob Halliwell, Richard B Gearry, Graham Radford-Smith, Jane M Andrews, Patrick C McHugh, Murray L Barclay
Background : Up to 20% of patients with inflammatory bowel disease (IBD) who are refractory to thiopurine therapy preferentially produce 6-methylmercaptopurine (6-MMP) at the expense of 6-thioguanine nucleotides (6-TGN), resulting in a high 6-MMP:6-TGN ratio (>20). The objective of this study was to evaluate whether genetic variability in guanine monophosphate synthetase (GMPS) contributes to preferential 6-MMP metabolizer phenotype. Methods: Exome sequencing was performed in a cohort of IBD patients with 6-MMP:6-TGN ratios of >100 to identify nonsynonymous single nucleotide polymorphisms (nsSNPs)...
May 16, 2018: Inflammatory Bowel Diseases
https://www.readbyqxmd.com/read/29786899/improved-stable-indocyanine-green-icg-mediated-cancer-optotheranostics-with-naturalized-hepatitis-b-core-particles
#16
Wenjun Shan, Ronghe Chen, Qiang Zhang, Jie Zhao, Binbin Chen, Xi Zhou, Shefang Ye, Shengli Bi, Liming Nie, Lei Ren
In recent years, hepatitis B core protein virus-like particle (HBc VLP) is an impressive biomaterial, which has attracted considerable attention due to favorable properties such as structural stability, high uptake efficiency, and biocompatibility in biomedical applications. Heretofore, only a few attempts have been made to apply it in physical, chemical, and biological therapy for cancer. In this study, a tumor-targeting RGD-HBc VLP is first fabricated through genetic engineering. For image-guided cancer phototherapy, indocyanine green (ICG) is loaded into RGD-HBc VLP via a disassembly/reassembly pathway and electrostatic attraction with high efficiency...
May 22, 2018: Advanced Materials
https://www.readbyqxmd.com/read/29786548/designer-human-tissue-coming-to-a-lab-near-you
#17
David C Hay, Cliona O'Farrelly
Human pluripotent stem cells (PSCs) offer a scalable alternative to primary and transformed human tissue. PSCs include human embryonic stem cells, derived from the inner cell mass of blastocysts unsuitable for human implantation; and induced PSCs, generated by the reprogramming of somatic cells. Both cell types display the ability to self-renew and retain pluripotency, promising an unlimited supply of human somatic cells for biomedical application. A distinct advantage of using PSCs is the ability to select for genetic background, promising personalized modelling of human biology 'in a dish' or immune-matched cell-based therapies for the clinic...
July 5, 2018: Philosophical Transactions of the Royal Society of London. Series B, Biological Sciences
https://www.readbyqxmd.com/read/29785653/valproic-acid-treatment-response-in-vitro-is-determined-by-tp53-status-in-medulloblastoma
#18
Bruna Mascaro-Cordeiro, Indhira Dias Oliveira, Francine Tesser-Gamba, Lorena Favaro Pavon, Nasjla Saba-Silva, Sergio Cavalheiro, Patrícia Dastoli, Silvia Regina Caminada Toledo
PURPOSE: Histone deacetylate inhibitors (HDACi), as valproic acid (VA), have been reported to enhance efficacy and to prevent drug resistance in some tumors, including medulloblastoma (MB). In the present study, we investigated VA role, combined to cisplatin (CDDP) in cell viability and gene expression of MB cell lines. METHODS: Dose-response curve determined IC50 values for each treatment: (1) VA single, (2) CDDP single, and (3) VA and CDDP combined. Cytotoxicity and flow cytometry evaluated cell viability after exposure to treatments...
May 22, 2018: Child's Nervous System: ChNS: Official Journal of the International Society for Pediatric Neurosurgery
https://www.readbyqxmd.com/read/29785024/targeting-skeletal-endothelium-to-ameliorate-bone-loss
#19
Ren Xu, Alisha Yallowitz, An Qin, Zhuhao Wu, Dong Yeon Shin, Jung-Min Kim, Shawon Debnath, Gang Ji, Mathias P Bostrom, Xu Yang, Chao Zhang, Han Dong, Pouneh Kermani, Sarfaraz Lalani, Na Li, Yifang Liu, Michael G Poulos, Amanda Wach, Yi Zhang, Kazuki Inoue, Annarita Di Lorenzo, Baohong Zhao, Jason M Butler, Jae-Hyuck Shim, Laurie H Glimcher, Matthew B Greenblatt
Recent studies have identified a specialized subset of CD31hi endomucinhi (CD31hi EMCNhi ) vascular endothelium that positively regulates bone formation. However, it remains unclear how CD31hi EMCNhi endothelium levels are coupled to anabolic bone formation. Mice with an osteoblast-specific deletion of Shn3, which have markedly elevated bone formation, demonstrated an increase in CD31hi EMCNhi endothelium. Transcriptomic analysis identified SLIT3 as an osteoblast-derived, SHN3-regulated proangiogenic factor...
May 21, 2018: Nature Medicine
https://www.readbyqxmd.com/read/29785010/genome-wide-analyses-identify-68-new-loci-associated-with-intraocular-pressure-and-improve-risk-prediction-for-primary-open-angle-glaucoma
#20
Anthony P Khawaja, Jessica N Cooke Bailey, Nicholas J Wareham, Robert A Scott, Mark Simcoe, Robert P Igo, Yeunjoo E Song, Robert Wojciechowski, Ching-Yu Cheng, Peng T Khaw, Louis R Pasquale, Jonathan L Haines, Paul J Foster, Janey L Wiggs, Chris J Hammond, Pirro G Hysi
Glaucoma is the leading cause of irreversible blindness globally 1 . Despite its gravity, the disease is frequently undiagnosed in the community 2 . Raised intraocular pressure (IOP) is the most important risk factor for primary open-angle glaucoma (POAG)3,4 . Here we present a meta-analysis of 139,555 European participants, which identified 112 genomic loci associated with IOP, 68 of which are novel. These loci suggest a strong role for angiopoietin-receptor tyrosine kinase signaling, lipid metabolism, mitochondrial function and developmental processes underlying risk for elevated IOP...
May 21, 2018: Nature Genetics
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