Medicine by Sfakianakis Alexandros: 05/07/17

Conditions: First Line Chemotherapy; Capecitabine Plus Cisplatin Versus Capecitabine Plus Paclitaxel; Advanced or Recurrent Esophageal Squamous Cell Carcinoma
Intervention: Drug: Capecitabine plus cisplatin(XP) versus capecitabine plus paclitaxel(XT)
Sponsor: Samsung Medical Center
Recruiting - verified April 2017

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Estradiol deficiency is a risk factor for idiopathic benign paroxysmal positional vertigo in postmenopausal female patients

Objectives/Hypothesis

Although it is generally considered that benign paroxysmal positional vertigo (BPPV) is associated with changes in female sex hormone levels, no direct data have been reported until now. The purpose of this article was to provide direct data showing the distinct relationship between female sex hormone fluctuations and BPPV in postmenopausal female patients.

Study Design

Prospective analysis in humans and basic research in animals.

Methods

Blood samples were analyzed to determine the levels of estradiol, progesterone, follicle-stimulating hormone, and luteinizing hormone in 50- to 80-year-old postmenopausal female patients newly diagnosed with idiopathic BPPV based on history compatible with BPPV and positive provocative maneuvers. Animal models of bilateral ovariectomy and female sex hormone replacement therapy were used to further confirm the relationship between BPPV and female sex hormone levels by determining the expression levels of otoconin 90, the protein suggested as essential in the dislocation of otoconia.

Results

Statistically significant differences between the estradiol level of BPPV patients and the control group were found (P < .001). Moreover, in bilateral ovariectomy in rats, 17β-estradiol replacement reversed the decrease of otoconin 90 levels.

Conclusions

Our results suggest that estradiol deficiency may be an important risk factor for idiopathic benign paroxysmal positional vertigo in postmenopausal female patients.

Level of Evidence

NA Laryngoscope, 2017

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In response to “Growth factors expression in hyaluronic acid fat graft myringoplasty”

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Evaluation of airway protection: Quantitative timing measures versus penetration/aspiration score

Objectives/Hypothesis

Quantitative measures of swallowing function may improve the reliability and accuracy of modified barium swallow (MBS) study interpretation. Quantitative study analysis has not been widely instituted, however, secondary to concerns about the time required to make measures and a lack of research demonstrating impact on MBS interpretation. This study compares the accuracy of the penetration/aspiration (PEN/ASP) scale (an observational visual-perceptual assessment tool) to quantitative measures of airway closure timing relative to the arrival of the bolus at the upper esophageal sphincter in identifying a failure of airway protection during deglutition.

Study Design

Retrospective review of clinical swallowing data from a university-based outpatient clinic.

Methods

Swallowing data from 426 patients were reviewed. Patients with normal PEN/ASP scores were identified, and the results of quantitative airway closure timing measures for three liquid bolus sizes were evaluated. The incidence of significant airway closure delay with and without a normal PEN/ASP score was determined. Inter-rater reliability for the quantitative measures was calculated.

Results

In patients with a normal PEN/ASP score, 33% demonstrated a delay in airway closure on at least one swallow during the MBS study. There was no correlation between PEN/ASP score and airway closure delay. Inter-rater reliability for the quantitative measure of airway closure timing was nearly perfect (intraclass correlation coefficient = 0.973).

Conclusions

The use of quantitative measures of swallowing function, in conjunction with traditional visual perceptual methods of MBS study interpretation, improves the identification of airway closure delay, and hence, potential aspiration risk, even when no penetration or aspiration is apparent on the MBS study.

Level of Evidence

4. Laryngoscope, 2017

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A randomized controlled trial of Velcro versus standard twill ties following pediatric tracheotomy

Objectives/Hypothesis

Tracheotomy is a common procedure. A reliable method of securing the tracheotomy tube is essential to minimize accidental decannulation. However, skin breakdown has been reported in ∼30% of patients. We sought to evaluate rates of skin-related complications and accidental decannulation with the use of Velcro ties compared to twill ties.

Study Design

A nonblinded, randomized controlled trial comparing Velcro versus twill ties in patients undergoing tracheotomy between July 1, 2014 and January 31, 2016.

Methods

Patients ≤21 years of age were recruited and randomized to receive either Velcro or twill ties. The primary outcome measure was skin-related complications. The secondary outcome measure was accidental decannulation. Outcome measures were followed through postoperative day 5.

Results

Ninety-three patients were eligible, and 63 were enrolled. No patients were withdrawn. Fifty-seven patients were included in the analysis. Twenty-seven (47.4%) received Velcro, and 30 (52.6%) received twill. Five enrolled patients did not undergo tracheotomy (one Velcro, four twill). One was diagnosed with a genetic skin condition after enrollment but prior to undergoing tracheotomy. Patient characteristics were similar between groups. No significant differences were found with respect to skin-related complications (P = .59). Six patients (20%) with twill ties required early tie change compared to two (7.4%) with Velcro ties (P = .5). Two accidental decannulations occurred in the twill group compared to one in the Velcro group (P = 1.0).

Conclusions

Our study demonstrated no differences in skin-related complications or accidental decannulation between Velcro and twill tracheotomy ties in the immediate postoperative period following tracheotomy. Our study suggests that Velcro ties are a viable alternative to twill ties.

Level of Evidence

1b Laryngoscope, 2017

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Sialendoscopy-assisted transfacial removal of parotid sialoliths: A systematic review and meta-analysis

Objective

To review the literature and conduct a meta-analysis to determine the effectiveness and safety of the combined endoscopic–transfacial approach for parotid sialolith management.

Data Sources

PubMed 1946–, Embase 1947–, Cumulative Index to Nursing and Allied Health Literature, the Cochrane Database of Systematic Reviews, Cochrane Database of Abstracts of Review Effects, Cochrane Central Register of Controlled Trials, clinicaltrials.gov, Proquest Dissertations and Theses, and FirstSearch Proceedings to March 2015.

Review Methods

Published prospective or retrospective English-language studies with reported outcomes of more than one patient undergoing the combined endoscopic-transfacial procedure for parotid sialolithiasis were included. Two independent authors screened all eligible studies and reviewed and extracted data from relevant publications. Weighted pooled proportions for stone removal, symptom improvement, gland preservation, and complications were calculated.

Results

Ten studies, primarily retrospective single-institution studies, were included in the final analysis, with a total of 184 patients. Overall, the procedure was noted to be successful with low risk; the weighted pooled proportions were 0.99 (95% confidence interval [CI]: 0.97 to 1.00) for stone removal, 0.97 (95% CI: 0.93 to 0.99) for symptom improvement, 1 (95% CI: 0.99 to 1.00) for gland preservation, and 0.06 (95% CI: 0.01 to 0.15) for complications.

Conclusions

Although our analysis is primarily based on retrospective data, the evidence shown here suggests that the combined endoscopic–transfacial technique is an effective treatment for parotid gland sialolithiasis not amenable to intraoral or purely endoscopic removal. This approach results in high rates of symptom improvement and gland preservation. The complication rates are low, further supporting the use of this technique. Laryngoscope, 2017

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The feasibility of gamma radiation sterilization for decellularized tracheal grafts

Objectives/Hypothesis

The most promising stem cell-derived tracheal transplantation approach is dependent upon the use of decellularized tracheal grafts. It has been assumed that a sterilization step, such as gamma radiation, would damage the delicate extracellular matrix of the graft, thus rendering it less viable for cellular repopulation, although this has not been thoroughly investigated.

Study Design

Laboratory-based comparative analysis.

Methods

Fifteen murine tracheas of strain C57/B-6 mice were obtained. Thirteen were subjected to a detergent-enzymatic decellularization process. Of these decellularized tracheas (DT), eight were irradiated, exposing five tracheas to a radiation level of 25 kGy (DT25) and three to 5 kGy (DT5). Two were left untreated. The two untreated tracheas, two DTs, and two DT25s were prepared and examined using both scanning and transmission electron microscopy. Bioburden calculations were obtained from three DTs, three DT25s, and three DT5s by homogenization, serial dilution, and streak plating.

Results

Electron microscopy of untreated fresh tracheas and DTs showed a slight qualitative degradation of cartilage ultrastructure due to the decellularization process. In contrast, examination of DT25 shows significant degradation including poor overall preservation of cartilage architecture with disorganized collagen fibers. The nonirradiated DTs had a calculated bacterial bioburden of 7.8 × 10⁷ to 3.4 × 10⁸ colony-forming units per gram. Both the DT25 and DT5 specimens were found to have a bioburden of zero.

Conclusions

Gamma radiation at 25 kGy degrades the architecture of decellularized tracheal grafts. These ultrastructural changes may prove detrimental to graft viability; however, bioburden calculations suggest that a 5 kGy radiation dose may be sufficient for sterilization.

Level of Evidence

NA Laryngoscope, 2017

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Health-related quality of life in patients with cleft palate: Validity and reliability of the VPI Effects on Life Outcomes (VELO) questionnaire translated to Dutch

Publication date: July 2017
Source:International Journal of Pediatric Otorhinolaryngology, Volume 98
Author(s): Laura Bruneel, Kristiane Van Lierde, Kim Bettens, Paul Corthals, Esther Van Poel, Evelien De Groote, Hannah Keppler
BackgroundDisease-specific health-related quality of life (HRQOL) questionnaires provide the clinician with important information regarding the impact of the disease on functioning and well-being. For patients with velopharyngeal insufficiency (VPI), the VPI Effects on Life Outcomes (VELO) questionnaire was developed and validated in English by Skirko et al. (2012). However, a valid and reliable Dutch translation of this questionnaire is not available yet.MethodsThe English questionnaire was translated to Dutch following a forward-backward translation procedure. A linguistic validation and the evaluation of the internal consistency (Cronbach's α) of this Dutch version were performed based on the responses of 39 parents of patients with cleft (lip and) palate (mean age: 6.8 years) (parent report) and the responses of 14 patients older than 8 years (mean age: 9.5 years) (child report). Additionally, the concurrent validity was assessed by comparing the scores on the parent report to those on the pediatric voice handicap index. Furthermore, the validity of the parent proxy assessment and the relationship between age and responses on the VELO questionnaire were investigated. Based on the responses of an age and gender matched control group without cleft palate, the discriminant validity was evaluated.ResultsThe parent report was easy to complete for all parents. Nine of the fourteen (64%) patients were able to complete the child report independently. The median scores on the parent report and the child report were 82.7 and 95.1 respectively. The patient group had a significantly worse perception of HRQOL compared to the control group (p < 0.001; p = 0.029). There were no significant differences between the responses of the parent and their child's (p = 0.345). A significant positive correlation was found between the score on the parent report and the age of the patients (p = 0.001). Furthermore, a significant negative correlation was found between the parent report and the P-VHI (p < 0.001). Cronbach's α was 0.955 and 0.817 for the parent report and the child report respectively.ConclusionThe Dutch VELO questionnaire is a valid, reliable and user-friendly tool that provides important information about HRQOL in patients with cleft (lip and) palate.

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An evaluation of the TREC assay with regard to the integration of SCID screening into the Dutch newborn screening program

Publication date: Available online 6 May 2017
Source:Clinical Immunology
Author(s): Maartje Blom, Ingrid Pico-Knijnenburg, Marja Sijne-van Veen, Anita Boelen, Robbert Bredius, Mirjam van der Burg, Peter Schielen
Newborn screening of severe combined immunodeficiency through the detection of T-cell receptor excision circles will provide the opportunity of treating before the occurrence of life-threatening infections. With the EnLite Neonatal TREC assay (PerkinElmer) and end-point PCR, 39 samples (3.0%) of 1295 heel prick cards of the Dutch newborn screening program required a retest after initial analysis. After retest, 21 samples (1.62%) gave TREC levels below cut-off. A significant reduction in TREC levels was observed in heel prick cards stored for three months (n=33) and one year (n=33). Preterm newborns (n=155) showed significantly lower TREC levels and a higher retest-rate than full-term newborns. Peripheral blood spots of 22 confirmed SCID patients and 17 primary immunodeficiency patients showed undetectable or low TREC-levels. These findings suggest that the EnLite Neonatal TREC assay is a suitable method for SCID-screening in the Netherlands, thereby providing guidance in the decisions concerning implementation into the Dutch program.

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Recurrent respiratory tract infections (RRTI) in the elderly: A late onset mild immunodeficiency?

Publication date: Available online 6 May 2017
Source:Clinical Immunology
Author(s): Esther van de Vosse, Monique M. van Ostaijen-ten Dam, René Vermaire, Els M. Verhard, Jacqueline L. Waaijer, Jaap A. Bakker, Sandra T. Bernards, Hermann Eibel, Maarten J. van Tol, Jaap T. van Dissel, Margje H. Haverkamp
Elderly with late-onset recurrent respiratory tract infections (RRTI) often have specific anti-polysaccharide antibody deficiency (SPAD). We hypothesized that late-onset RRTI is caused by mild immunodeficiencies, such as SPAD, that remain hidden through adult life. We analyzed seventeen elderly RRTI patients and matched controls. We determined lymphocyte subsets, expression of BAFF receptors, serum immunoglobulins, complement pathways, Pneumovax-23 vaccination response and genetic variations in BAFFR and MBL2. Twelve patients (71%) and ten controls (59%) had SPAD. IgA was lower in patients than in controls, but other parameters did not differ. However, a high percentage of both patients (53%) and controls (65%) were MBL deficient, much more than in the general population. Often, MBL2 secretor genotypes did not match functional deficiency, suggesting that functional MBL deficiency can be an acquired condition. In conclusion, we found SPAD and MBL deficiency in many elderly, and conjecture that at least the latter arises with age.

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Objective Bayesian Survival Analysis Using Shape Mixtures of Log-Normal Distributions

Vallejos, CA; Steel, MFJ; (2015) Objective Bayesian Survival Analysis Using Shape Mixtures of Log-Normal Distributions. Journal of the American Statistical Association , 110 (510) pp. 697-710. 10.1080/01621459.2014.923316 .

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Incorporating unobserved heterogeneity in Weibull survival models: A Bayesian approach

Vallejos, CA; Steel, MFJ; (2017) Incorporating unobserved heterogeneity in Weibull survival models: A Bayesian approach. Econometrics and Statistics 10.1016/j.ecosta.2017.01.005 .

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Beyond comparisons of means: understanding changes in gene expression at the single-cell level

Vallejos, CA; Richardson, S; Marioni, JC; (2016) Beyond comparisons of means: understanding changes in gene expression at the single-cell level. Genome Biology , 17 (1) 10.1186/s13059-016-0930-3 .

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BASiCS: Bayesian Analysis of Single-Cell Sequencing Data.

Vallejos, CA; Marioni, JC; Richardson, S; (2015) BASiCS: Bayesian Analysis of Single-Cell Sequencing Data. PLoS Comput Biol , 11 (6) , Article e1004333. 10.1371/journal.pcbi.1004333 .

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Aging increases cell-to-cell transcriptional variability upon immune stimulation

Martinez-Jimenez, CP; Eling, N; Chen, H-C; Vallejos, CA; Kolodziejczyk, AA; Connor, F; Stojic, L; Martinez-Jimenez, CP; Eling, N; Chen, H-C; Vallejos, CA; Kolodziejczyk, AA; Connor, F; Stojic, L; Rayner, TF; Stubbington, MJT; Teichmann, SA; de la Roche, M; Marioni, JC; Odom, DT; - view fewer (2017) Aging increases cell-to-cell transcriptional variability upon immune stimulation. Science , 355 (6332) pp. 1433-1436. 10.1126/science.aah4115 .

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THE STATUS OF YOHKOH IN ORBIT - AN INTRODUCTION TO THE INITIAL SCIENTIFIC RESULTS

OGAWARA, Y; ACTON, LW; BENTLEY, RD; BRUNER, ME; CULHANE, JL; HIEI, E; HIRAYAMA, T; OGAWARA, Y; ACTON, LW; BENTLEY, RD; BRUNER, ME; CULHANE, JL; HIEI, E; HIRAYAMA, T; HUDSON, HS; KOSUGI, T; LEMEN, JR; STRONG, KT; TSUNETA, S; UCHIDA, Y; WATANABE, T; YOSHIMORI, M; - view fewer (1992) THE STATUS OF YOHKOH IN ORBIT - AN INTRODUCTION TO THE INITIAL SCIENTIFIC RESULTS. PUBLICATIONS OF THE ASTRONOMICAL SOCIETY OF JAPAN , 44 (5) L41-+.

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Specificity, Privacy, and Degeneracy in the CD4 T Cell Receptor Repertoire Following Immunization.

Sun, Y; Best, K; Cinelli, M; Heather, JM; Reich-Zeliger, S; Shifrut, E; Friedman, N; Sun, Y; Best, K; Cinelli, M; Heather, JM; Reich-Zeliger, S; Shifrut, E; Friedman, N; Shawe-Taylor, J; Chain, B; - view fewer (2017) Specificity, Privacy, and Degeneracy in the CD4 T Cell Receptor Repertoire Following Immunization. Front Immunol , 8 430-. 10.3389/fimmu.2017.00430 .

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Investments in cancer research awarded to UK institutions and the global burden of cancer 2000-2013: a systematic analysis.

Maruthappu, M; Head, MG; Zhou, CD; Gilbert, BJ; El-Harasis, MA; Raine, R; Fitchett, JR; Maruthappu, M; Head, MG; Zhou, CD; Gilbert, BJ; El-Harasis, MA; Raine, R; Fitchett, JR; Atun, R; - view fewer (2017) Investments in cancer research awarded to UK institutions and the global burden of cancer 2000-2013: a systematic analysis. BMJ Open , 7 (4) , Article e013936. 10.1136/bmjopen-2016-013936 .

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The UK Myotonic Dystrophy Patient Registry: facilitating and accelerating clinical research.

Wood, L; Cordts, I; Atalaia, A; Marini-Bettolo, C; Maddison, P; Phillips, M; Roberts, M; Wood, L; Cordts, I; Atalaia, A; Marini-Bettolo, C; Maddison, P; Phillips, M; Roberts, M; Rogers, M; Hammans, S; Straub, V; Petty, R; Orrell, R; Monckton, DG; Nikolenko, N; Jimenez-Moreno, AC; Thompson, R; Hilton-Jones, D; Turner, C; Lochmüller, H; - view fewer (2017) The UK Myotonic Dystrophy Patient Registry: facilitating and accelerating clinical research. J Neurol 10.1007/s00415-017-8483-2 .

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Could thirst-triggered self-administration of intravenous fluid lead to more rapid rehydration than clinician-directed infusion?

Hughes, FE; Mythen, MG; Montgomery, HE; (2017) Could thirst-triggered self-administration of intravenous fluid lead to more rapid rehydration than clinician-directed infusion? In: (Proceedings) Annual Meeting of the Society-of-Academic-and-Research-Surgery (SARS). (pp. p. 62). WILEY

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Comparing Submillimeter Polarized Emission with Near-infrared Polarization of Background Stars for the Vela C Molecular Cloud

Santos, FP; Ade, PAR; Angile, FE; Ashton, P; Benton, SJ; Devlin, MJ; Dober, B; Santos, FP; Ade, PAR; Angile, FE; Ashton, P; Benton, SJ; Devlin, MJ; Dober, B; Fissel, LM; Fukui, Y; Galitzki, N; Gandilo, NN; Klein, J; Korotkov, AL; Li, Z-Y; Martin, PG; Matthews, TG; Moncelsi, L; Nakamura, F; Netterfield, CB; Novak, G; Pascale, E; Poidevin, F; Savini, G; Scott, D; Shariff, JA; Soler, JD; Thomas, NE; Tucker, CE; Tucker, GS; Ward-Thompson, D; - view fewer (2017) Comparing Submillimeter Polarized Emission with Near-infrared Polarization of Background Stars for the Vela C Molecular Cloud. ASTROPHYSICAL JOURNAL , 837 (2) 10.3847/1538-4357/aa62a7 .

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Chiari Malformation Type 1: A Systematic Review of Natural History and Conservative Management.

Langridge, B; Phillips, E; Choi, D; (2017) Chiari Malformation Type 1: A Systematic Review of Natural History and Conservative Management. World Neurosurg 10.1016/j.wneu.2017.04.082 .

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Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.

Debnath, S; Jaako, P; Siva, K; Rothe, M; Chen, J; Dahl, M; Gaspar, HB; Debnath, S; Jaako, P; Siva, K; Rothe, M; Chen, J; Dahl, M; Gaspar, HB; Flygare, J; Schambach, A; Karlsson, S; - view fewer (2017) Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia. Mol Ther 10.1016/j.ymthe.2017.04.002 .

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Reproducibility, and sensitivity to motor unit loss in amyotrophic lateral sclerosis, of a novel MUNE method: MScanFit MUNE.

Jacobsen, AB; Bostock, H; Fuglsang-Frederiksen, A; Duez, L; Beniczky, S; Møller, AT; Blicher, JU; Jacobsen, AB; Bostock, H; Fuglsang-Frederiksen, A; Duez, L; Beniczky, S; Møller, AT; Blicher, JU; Tankisi, H; - view fewer (2017) Reproducibility, and sensitivity to motor unit loss in amyotrophic lateral sclerosis, of a novel MUNE method: MScanFit MUNE. Clin Neurophysiol 10.1016/j.clinph.2017.03.045 .

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DynaMoVis: visualization of dynamic models for urban modeling

Dearden, J; Jones, MW; Wilson, A; (2015) DynaMoVis: visualization of dynamic models for urban modeling. VISUAL COMPUTER , 31 (6-8) pp. 1079-1088. 10.1007/s00371-015-1096-9 .

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Complementary feeding in preterm infants

Embleton, ND; Fewtrell, M; (2017) Complementary feeding in preterm infants. LANCET GLOBAL HEALTH , 5 (5) E470-E471.

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Child maltreatment and living standards in mid-adulthood: findings from the 1958 British birth cohort

Pereira, SMP; Li, L; Power, C; (2016) Child maltreatment and living standards in mid-adulthood: findings from the 1958 British birth cohort. In: OXFORD UNIV PRESS

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COMPARISON OF NEXT GENERATION SEQUENCING TECHNOLOGIES FOR THE COMPREHENSIVE ASSESSMENT OF FULL-LENGTH HEPATITIS C VIRAL GENOMES

Thomson, E; Ip, C; Badhan, A; Christiansen, M; Adamson, W; Ansari, A; Bibby, D; Thomson, E; Ip, C; Badhan, A; Christiansen, M; Adamson, W; Ansari, A; Bibby, D; Bonsall, D; Bowden, R; Breuer, J; Brown, A; Bryant, J; Filipe, ADS; Hinds, C; Hudson, E; Klenerman, P; Lythgow, K; Mbisa, J; McLauchlan, J; Myers, R; Piazza, P; Roy, S; Trebes, A; Vattipally, S; Witteveldt, J; Simmonds, P; Barnes, E; - view fewer (2016) COMPARISON OF NEXT GENERATION SEQUENCING TECHNOLOGIES FOR THE COMPREHENSIVE ASSESSMENT OF FULL-LENGTH HEPATITIS C VIRAL GENOMES. In: (Proceedings) EASL International Liver Congress. (pp. S415-S415). ELSEVIER SCIENCE BV

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Improving genetic diagnosis in Mendelian disease with transcriptome sequencing

Cummings, BB; Marshall, JL; Tukiainen, T; Lek, M; Donkervoort, S; Foley, AR; Bolduc, V; Cummings, BB; Marshall, JL; Tukiainen, T; Lek, M; Donkervoort, S; Foley, AR; Bolduc, V; Waddell, LB; Sandaradura, SA; O'Grady, GL; Estrella, E; Reddy, HM; Zhao, F; Weisburd, B; Karczewski, KJ; O'Donnell-Luria, AH; Birnbaum, D; Sarkozy, A; Hu, Y; Gonorazky, H; Claeys, K; Joshi, H; Bournazos, A; Oates, EC; Ghaoui, R; Davis, MR; Laing, NG; Topf, A; Kang, PB; Beggs, AH; North, KN; Straub, V; Dowling, JJ; Muntoni, F; Clarke, NF; Cooper, ST; Bonnemann, CG; MacArthur, DG; - view fewer (2017) Improving genetic diagnosis in Mendelian disease with transcriptome sequencing. SCIENCE TRANSLATIONAL MEDICINE , 9 (386) 10.1126/scitranslmed.aa15209 .

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In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.

Mattar, CNZ; Gil-Farina, I; Rosales, C; Johana, N; Tan, YYW; McIntosh, J; Kaeppel, C; Mattar, CNZ; Gil-Farina, I; Rosales, C; Johana, N; Tan, YYW; McIntosh, J; Kaeppel, C; Waddington, SN; Biswas, A; Choolani, M; Schmidt, M; Nathwani, AC; Chan, JKY; - view fewer (2017) In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model. Mol Ther 10.1016/j.ymthe.2017.04.003 .

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Κυριακή 7 Μαΐου 2017

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