Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia.

autologous hematopoietic stem cell lentiviral-based gene therapy with betibeglogene autotemcel has been studied in patients with transfusion-dependent β-thalassemia in Phase III clinical trials (HGB-207 and HGB-212), with 90% of patients reaching transfusion independence (TI). Here, we explore manufacturing parameters, drug product quality attributes, and limited patient characteristics that had an impact on clinical efficacy in HGB-207 and HGB-212. Retrospective analysis revealed that the peripheral blood vector copy number (VCN) was related to TI, with a strong correlation between peripheral blood VCN at 6 months and gene therapy-derived therapeutic protein (HbA) expression at 6 months (correlation coefficient, 0.

ACVR and ECVDI consensus statement for the standardization of the abdominal ultrasound examination.

This consensus statement is designed to provide a standard of care document and describes the ACVR and ECVDI definition for performing a standard abdominal ultrasound examination in dogs and cats. The ACVR and ECVDI define a standard abdominal ultrasonographic examination as a complete exam of the abdominal organs which is appropriately documented. The consensus statement intends to provide guidance to veterinary sonographers and veterinarians for the performance and documentation of high-quality diagnostic ultrasound examination.

Diagnostic imaging for the assessment of acquired abdominal vascular diseases in small animals: A pictorial review.

Advances in interventional radiology and surgical techniques now allow complex abdominal diseases to be more successfully treated in small animals. Abdominal vascular alterations, acquired as individual process or as complication of other lesions such as neoplasia, can be life-threatening or at least greatly limit curative interventions of underlying diseases. Computed tomography (CT) and high-definition ultrasonography are now readily available in veterinary referral centers.

Management of drooling in children with cerebral palsy: A French survey.

Aim: To characterise children with cerebral palsy (CP) and pathological drooling in France, and to describe care pathways, assessment and treatment.

Method: A transversal, observational, descriptive survey of the practices and opinions of 400 health professionals potentially involved in the care of children with CP, was carried out nationally across France in 2013.

Results: The response rate was 36%.

Ultrasonographic and clinicopathological features of pyloroduodenal adenomatous polyps in cats.

Objectives The aim of the study was to report the ultrasonographic and clinicopathological findings in cats with confirmed pyloroduodenal adenomatous polyps. Methods Clinicopathological data, ultrasonographic and histopathological findings were collected retrospectively from medical records. Results Pyloroduodenal polyps appeared as small moderately echogenic and homogeneous nodules filling most of the proximal duodenal or pyloroduodenal lumen.

Ileocecocolic volvulus in a German shepherd dog.

This report describes an ileocecocolic volvulus in a German shepherd dog with risk factors of previous abdominal surgeries and concurrent chronic enteropathy. Contrast-enhanced computed tomography (CT) with multiplanar reformatting was more sensitive than abdominal radiographs or ultrasound to obtain a diagnosis, because of the presence of a "whirl-sign" on CT. A combination of colopexy and cecopexy was succesfully used to treat the patient's condition.

Maximizing recombinant human serum albumin production in a Mut(s) Pichia pastoris strain.

Human serum albumin (HSA) is a cysteine rich molecule that is most abundant in human blood plasma. To remain viable in the market due to lower marketing costs for HSA, it is important to produce a large quantity in an economical manner by recombinant technology. The objective of this study was to maximize recombinant HSA (rHSA) production using a Mut(s) Pichia pastoris strain by fermentation process optimization.

Relevance of intraglandular injections of Botulinum toxin for the treatment of sialorrhea in children with cerebral palsy: a review.

Background: After the age of 4 years, drooling becomes pathological and impacts the quality of life of children with cerebral palsy. Intraglandular injection of Botulinum toxin is one of the treatments available to limit this phenomenon.

Aims: The objectives of this review were to validate the efficacy of Botulinum toxin injections for drooling in children with cerebral palsy, determine recommendations and identify potential side effects.

Computed tomographic characteristics of collateral venous pathways in dogs with caudal vena cava obstruction.

Collateral venous pathways develop in dogs with obstruction or increased blood flow resistance at any level of the caudal vena cava in order to maintain venous drainage to the right atrium. The purpose of this retrospective study was to describe the sites, causes of obstruction, and configurations of venous collateral pathways for a group of dogs with caudal vena cava obstruction. Computed tomography databases from two veterinary hospitals were searched for dogs with a diagnosis of caudal vena cava obstruction and multidetector row computed tomographic angiographic (CTA) scans that included the entire caudal vena cava.

Radiographic diagnosis of mechanical obstruction in dogs based on relative small intestinal external diameters.

Mechanical obstruction is a frequent cause of acute vomiting in dogs requiring prompt diagnosis to improve patient management and prognosis. The purpose of this retrospective study was to compare small intestinal radiographic characteristics in dogs with versus without mechanical intestinal obstruction. Fifty dogs with gastrointestinal clinical signs and abdominal radiographs were recruited from hospital record archives and assigned to groups (group 1, obstructive, n = 25; group 2, nonobstructive n = 25).

A posteriori comparison of natural and surgical destabilization models of canine osteoarthritis.

For many years Canis familiaris, the domestic dog, has drawn particular interest as a model of osteoarthritis (OA). Here, we optimized the dog model of experimental OA induced by cranial cruciate ligament sectioning. The usefulness of noninvasive complementary outcome measures, such as gait analysis for the limb function and magnetic resonance imaging for structural changes, was demonstrated in this model.

Fast presurgical magnetic resonance imaging of meniscal tears and concurrent subchondral bone marrow lesions. Study of dogs with naturally occurring cranial cruciate ligament rupture.

Meniscal tears and subchondral bone marrow lesions have both been described in dogs with cranial cruciate ligament rupture, but their possible concurrence has not been evaluated. In a population of 14 dogs exhibiting signs of stifle pain with surgically confirmed cranial cruciate ligament rupture, a short presurgical 1.5T magnetic resonance (MR) imaging protocol including dorsal proton density, dorsal T1-weighted gradient recalled echo, and sagittal fat-saturated dual echo sequences was tested to further investigate these features and illustrate meniscal tears.

Validation of a novel technique for creating simulated radiographs using computed tomography datasets.

Understanding radiographic anatomy and the effects of varying patient and radiographic tube positioning on image quality can be a challenge for students. The purposes of this study were to develop and validate a novel technique for creating simulated radiographs using computed tomography (CT) datasets. A DICOM viewer (ORS Visual) plug-in was developed with the ability to move and deform cuboidal volumetric CT datasets, and to produce images simulating the effects of tube-patient-detector distance and angulation.

Regulation of alcohol oxidase 1 (AOX1) promoter and peroxisome biogenesis in different fermentation processes in Pichia pastoris.

Production of recombinant proteins is affected by process conditions, where transcriptional regulation of Pichia pastoris alcohol oxidase 1 (PpAOX1) promoter has been a key factor to influence expression levels of proteins of interest. Here, we demonstrate that the AOX1 promoter and peroxisome biogenesis are regulated based on different process conditions. Two types of GFP-fusion proteins, Ub-R-GFP (short-lived GFP in the cytosol) and GFP-SKL (peroxisomal targeting GFP), were successfully used to characterize the time-course of the AOX1 promoter and peroxisome biogenesis, respectively.

Interobserver agreement and diagnostic accuracy of brain magnetic resonance imaging in dogs.

Objective: To evaluate interobserver agreement and diagnostic accuracy of brain MRI in dogs.

Design: Evaluation study.

Animals: 44 dogs.

Ultrasound-guided percutaneous antegrade pyelography with computed tomography for the diagnosis of spontaneous partial ureteral rupture in a dog.

A 10-year-old spayed female dalmatian dog developed acute vomiting and abdominal pain. Ultrasound examination of the abdomen showed right hydronephrosis and proximal ureter dilation with mild retroperitoneal free fluid. Computed tomography (CT) of the abdomen confirmed the ultrasonographic findings and revealed, additionally, a right ureteral stone.

Improvement of N-glycan site occupancy of therapeutic glycoproteins produced in Pichia pastoris.

Yeast is capable of performing posttranslational modifications, such as N- or O-glycosylation. It has been demonstrated that N-glycans play critical biological roles in therapeutic glycoproteins by modulating pharmacokinetics and pharmacodynamics. However, N-glycan sites on recombinant glycoproteins produced in yeast can be underglycosylated, and hence, not completely occupied.

A comparison of computed tomography, computed radiography, and film-screen radiography for the detection of canine pulmonary nodules.

Computed tomography (CT) has become more widely available and computed radiography (CR) has replaced film-screen radiography for canine thoracic imaging in many veterinary practices. There are limited data comparing these modalities in a veterinary clinical setting to detect pulmonary nodules. We compared CT, CR, and film-screen radiography for detecting the presence, number, and characteristics of pulmonary nodules in dogs.

Evaluation of osteoarthritis in cats: novel information from a pilot study.

Objective: To describe structural changes associated with osteoarthritis (OA) in cats and to quantify OA-associated disability using functional evaluations.

Study Design: Cross-sectional pilot study with longitudinal data.

Animals: Normal cats (n = 2) and coxofemoral joint OA cats (n = 4) were evaluated by physical examination, radiography, and magnetic resonance imaging (MRI).

Gallbladder sludge on ultrasound is predictive of increased liver enzymes and total bilirubin in cats.

The purposes of this retrospective study were to assess the prevalence of gallbladder sludge (GBS) in a population of cats presented for abdominal ultrasound in a teaching hospital and to determine its association with increased serum alanine aminotransferase (ALT), alkaline phosphatase (ALP), and total bilirubin (TB). Gallbladder sludge was detected in 152 (14%) of the cats undergoing abdominal ultrasound between 2004 and 2008. This population was compared to a control group of 32 cats without GBS.

Costochondral junction osteomyelitis in 3 septic foals.

The costochondral junction constitutes a potential site of infection in septic foals and it could be favored by thoracic trauma. Standard radiographs and ultrasonography are useful tools for diagnosis of this condition and ultrasound-guided needle aspiration could permit the definitive confirmation of infection.

Palpation and dorsal acetabular rim radiographic projection for early detection of canine hip dysplasia: a prospective study.

Objective: To determine the value of 2 diagnostic methods: (1) the reduction angle (RA) using the Ortolani maneuver and (2) the dorsal acetabular slope (DAS) from the dorsal acetabular rim (DAR) radiographic projection, to predict osteoarthritis (OA) in dogs with hip dysplasia.

Study Design: Prospective study.

Sample Population: Dogs (n = 73).

Physical and rehabilitation medicine (PRM) care pathways: "stroke patients".

This document is part of a series of documents designed by the French Physical and Rehabilitation Medicine Society (SOFMER) and the French Federation of PRM (FEDMER). These documents describe the needs for a specific type of patients; PRM care objectives, human and material resources to be implemented, chronology as well as expected outcomes. "Care pathways in PRM" is a short document designed to enable the reader (physicians, decision-maker, administrator, lawyer or finance manager) to quickly apprehend the needs of these patients and the available therapeutic care structures for proper organization and pricing of these activities.

Optimization of erythropoietin production with controlled glycosylation-PEGylated erythropoietin produced in glycoengineered Pichia pastoris.

Pichia pastoris is a methylotropic yeast that has gained great importance as an organism for protein expression in recent years. Here, we report the expression of recombinant human erythropoietin (rhEPO) in glycoengineered P. pastoris.

Effect of acquisition time and chemical fat suppression on meningeal enhancement on MR imaging in dogs.

Our purpose was to characterize meningeal gadolinium enhancement on magnetic resonance (MR) imaging in dogs with inflammatory and neoplastic diseases, and to assess interobserver variability and the impact of delayed acquisition and chemical fat saturation on its conspicuity. Transverse T1-weighted FLAIR images were acquired prior to, and immediately following gadolinium injection (T0), and at 5 (T5) and 15-20 min delay (TD), with and without fat suppression, in 155 consecutive dogs imaged for suspected brain disease. The agreement on meningeal enhancement was globally substantial (kappa = 0.