Results 51 to 60 of about 79,600 (335)

Pathogenic Germline PALB2 and RAD50 Variants in Patients With Relapsed Ewing Sarcoma

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Approximately 10% of patients with Ewing sarcoma (EwS) have pathogenic germline variants. Here, we report two cases: first, a novel germline pathogenic variant in partner and localizer of BRCA2 (PALB2) in a patient with a late EwS relapse. Its impact on homologous recombination is demonstrated, and breast cancer risk is discussed.
Molly Mack, A. Carolina Tufino, Mohd Azrin Jamalruddin, Sreya Dey, Yinghong Pan, Elina Mukherjee, Jessica D. Daley, Julia Meade, Mahmoud Aarabi, Lisa Maurer, James Cooper, Kara A. Bernstein, Kelly M. Bailey   +12 more
wiley   +1 more source

Surrogate endpoints for overall survival in digestive oncology trials: which candidates? A questionnaires survey among clinicians and methodologists

BMC Cancer, 2010
Background Overall survival (OS) is the gold standard for the demonstration of a clinical benefit in cancer trials. Replacement of OS by a surrogate endpoint allows to reduce trial duration.
Bonnetain Franck, Bedenne Laurent, Methy Nicolas   +2 more
doaj   +1 more source

Comparing Biomarkers as Principal Surrogate Endpoints [PDF]

Biometrics, 2011
Recently a new definition of surrogate endpoint, the "principal surrogate," was proposed based on causal associations between treatment effects on the biomarker and on the clinical endpoint. Despite its appealing interpretation, limited research has been conducted to evaluate principal surrogates, and existing methods focus on risk models that consider
Huang, Ying, Gilbert, Peter B.
openaire   +3 more sources

Mapping the evolution of mitochondrial complex I through structural variation

FEBS Letters, EarlyView.
Respiratory complex I (CI) is crucial for bioenergetic metabolism in many prokaryotes and eukaryotes. It is composed of a conserved set of core subunits and additional accessory subunits that vary depending on the organism. Here, we categorize CI subunits from available structures to map the evolution of CI across eukaryotes. Respiratory complex I (CI)
Dong‐Woo Shin, Tingting Chen, James A. Letts   +2 more
wiley   +1 more source

Applications of neuroimaging to disease-modification trials in Alzheimer's disease. [PDF]

, 2009
Critical to development of new therapies for Alzheimer's disease (AD) is the ability to detect clinical or pathological change over time. Clinical outcome measures typically used in therapeutic trials have unfortunately proven to be relatively variable ...
Aisen, Paul S, Alzheimer's Disease Neuroimaging Initiative, Brewer, James B, Chen, Kewei, Donohue, Michael, Fleisher, Adam S   +5 more
core   +2 more sources

The anti‐CRISPR protein AcrIE8.1 inhibits the type I‐E CRISPR‐Cas system by directly binding to the Cascade subunit Cas11

FEBS Letters, EarlyView.
In this study, we present the structure of AcrIE8.1, a previously uncharacterized anti‐CRISPR protein that inhibits the type I‐E CRISPR‐Cas system. Through a combination of structural and biochemical analyses, we demonstrate that AcrIE8.1 directly binds to the Cas11 subunit of the Cascade complex to inhibit the CRISPR‐Cas system.
Young Woo Kang, Hyun Ho Park
wiley   +1 more source

Protocol for the development of SPIRIT and CONSORT extensions for randomised controlled trials with surrogate primary endpoints: SPIRIT-SURROGATE and CONSORT-SURROGATE

BMJ Open, 2022
Introduction Randomised controlled trials (RCTs) may use surrogate endpoints as substitutes and predictors of patient-relevant/participant-relevant final outcomes (eg, survival, health-related quality of life).
Martin Offringa, Nancy J Butcher, Derek Stewart, An-Wen Chan, Oriana Ciani, Philippa Davies, Christopher J Weir, Mario Ouwens, Dalia Dawoud, Sylwia Bujkiewicz, Anthony Muchai Manyara   +10 more
doaj   +1 more source

Use of surrogate endpoints in healthcare policy : proposal for consistent adoption of a validation framework [PDF]

, 2016
We propose a three step framework for the evaluation of surrogate endpoints for health policy decisions on health ...
Everardo D. Saad, FM Clement, Guido Rasi, HC Bucher, M Buyse, Marc Buyse, Mike Drummond, NS Downing, NS Downing, O Ciani, O Ciani, O Ciani, Oriana Ciani, Rod S. Taylor, T Burzykowski, TR Fleming   +15 more
core   +2 more sources

Surrogate Endpoints in Medicine [PDF]

Disease Markers, 2002
A surrogate endpoint is defined as a biomarker intended to substitute for a clinical endpoint, such as an incidence, or mortality. The evidence supporting the linkage of a biomarker to accurately substitute for a clinical endpoint may be derived from epidemiologic studies, clinical trials, in vitro analysis, animal models, and simulated biologic ...
Srivastava, Sudhir, Wagner, John A.
openaire   +1 more source

A Cre‐dependent lentiviral vector for neuron subtype‐specific expression of large proteins

FEBS Letters, EarlyView.
We designed a versatile and modular lentivector comprising a Cre‐dependent switch and self‐cleaving 2A peptide and tested it for co‐expression of GFP and a 2.8 kb gene of interest (GOI) in mouse cortical parvalbumin (PV+) interneurons and midbrain dopamine (TH+) neurons.
Weixuan Xue, Sandrine Picaud, Régine Hepp, Stéphanie Pons, Uwe Maskos, Bertrand Lambolez, Ludovic Tricoire   +6 more
wiley   +1 more source