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Gene Therapy for Hemophilia B: Achievements, Open Issues, and Perspectives.

Hemophilia B is the first bleeding disorder for which gene therapy clinical programs began. Presently, adenovirus-associated vectors represent the best means to deliver the transgene, and their administration by intravenous route has been used in recent clinical trials. The natural occurring factor IX (FIX) Padua variant, which allows for a 5- to 8-fold higher activity of FIX, while maintaining a normal protein concentration, was subsequently used to enhance the level of transgene expression. All the recent trials using this variant showed good results, and accumulating data suggest that long-

Achieving excellence with interdisciplinary approaches in complex orthodontic adult patients.

Over the last decades the percentage of adult orthodontic patients has substantially increased. Undeniably, an important motif for seeking orthodontic care at an older age is smile improvement, but this is not all. Frequently, impaired dentofacial aesthetics are combined with several other issues: severe dentoskeletal malocclusions; multiple tooth loss due to caries or endodontic failure; dental agenesis or trauma; periodontal breakdown; or functional problems, such as temporomandibular disorders or obstructive sleep apnoea. Therefore, comprehensive adult treatment mostly requires close collab

Lofgren Syndrome: Achieving an Accurate Diagnosis for Improved Patient Care.

Lofgren syndrome is a clinically distinct phenotype of sarcoidosis. It is characterized by the triad of bilateral hilar lymphadenopathy, arthritis (usually the ankles), and fever. We present the case of a 31-year-old male patient who presented with fever and edema in both lower limbs, with palpation of subcutaneous nodules. A chest contrast-enhanced computerized axial tomography (CECT) scan revealed perihilar and mediastinal lymphadenopathy. In making the diagnosis, tuberculosis and lymphoma were both ruled out. A mediastinoscopy confirmed Lofgren syndrome. In medicine, a good differential dia

Using construction procurement strategy to achieve socioeconomic development objectives.

The procurement strategy for a construction project should provide the framework to achieve secondary procurement and socio-economic development objectives [2]. However, little attention has been focused on this in theory and practice. This paper addresses that gap by presenting a case study of the innovative targeting strategy developed and successfully implemented on a New Universities Project in South Africa to promote specified socioeconomic development objectives. Document analysis was used to examine how four socioeconomic development targets or key performance indicators, namely: local

Achieving Cardiovascular Risk Management Goals and Patient Quality of Life.

(1) Background: Eliminating or reducing the severity of modifiable risk factors of cardiovascular disease (CVD) and undertaking health-promoting behaviors is the basis for prevention. (2) Methods: This study included 200 subjects without a history of CVD, aged 18 to 80 years, who had been diagnosed with hypertension, hypercholesterolemia, or diabetes 6 to 24 months before study enrolment. (3) Results: The median 10-year CV risk assessed by the SCORE2 and SCORE2-OP algorithms was 3.0 (IQR 1.5-7.0). An increase in mean cardiovascular risk in the range from low and moderate to very high was assoc

New Avenues and Major Achievements in Phytocompounds Research for Glioblastoma Therapy.

Phytocompounds have been evaluated for their anti-glioblastoma actions for decades, with promising results from preclinical studies but only limited translation into clinics. Indeed, by targeting multiple signaling pathways deregulated in cancer, they often show high efficacy in the in vitro studies, but their poor bioavailability, low tumor accumulation, and rapid clearance compromise their efficacy in vivo. Here, we present the new avenues in phytocompound research for the improvement of glioblastoma therapy, including the ways to enhance the response to temozolomide using phytochemicals, th

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… In this article, we present the most up-to-date revision of the new, promising avenues and major achievements in phytocompounds research for GBM therapy, bringing the development of efficacious anti-GBM treatments closer. … The authors utilized sophisticated computational tools, specifically PyRx and the AutoDock Vina wizard, to achieve a more detailed understanding and guarantee the accuracy of their findings. …

Bone ingrowth into a porous structure is achieved by preceding fibrogenesis and vascularization.

Porous structures are frequently used in surgical implants to strengthen the interlocking power produced by bone ingrowth. Therefore, we aimed to elucidate the mechanism underlying bone ingrowth into a porous structure accompanied by vascularization. A nonbioactive polyetheretherketone implant with a 3D-printed porous structure was prepared and implanted in a bone hole created in the tibias of rabbits. We observed bone ingrowth in the same individual specimens immediately and at 2, 4, 8, and 12 weeks post-implantation using in-vivo computed tomography (CT). Furthermore, a detailed evaluation w

How to achieve adequate quenching for DBP analysis in drinking water?

Quenching is an important step to terminate disinfection during preparation of disinfected water samples for the analysis of disinfection byproducts (DBPs). However, an incomplete quenching might result in continued reactions of residual chlorine, whereas an excessive quenching might decompose target DBPs. Therefore, an adequate quenching to achieve simultaneous disinfection termination and DBP preservation is of particular importance. In this study, the two-stage reaction kinetics of chlorine and three commonly used quenching agents (i.e., ascorbic acid, sodium thiosulfate, and sodium sulfite

Effectiveness of bracing to achieve curve regression in adolescent idiopathic scoliosis.

AIMS: To systematically evaluate whether bracing can effectively achieve curve regression in patients with adolescent idiopathic scoliosis (AIS), and to identify any predictors of curve regression after bracing. METHODS: Two independent reviewers performed a comprehensive literature search in PubMed, Ovid, Web of Science, Scopus, and Cochrane Library to obtain all published information about the effectiveness of bracing in achieving curve regression in AIS patients. Search terms included "brace treatment" or "bracing," "idiopathic scoliosis," and "curve regression" or "curve reduction." Inclu

The IBEX Knowledge-Base: Achieving more together with open science.

Iterative Bleaching Extends multipleXity (IBEX) is a versatile method for highly multiplexed imaging of diverse tissues. Based on open science principles, we created the IBEX Knowledge-Base, a resource for reagents, protocols and more, to empower innovation.

Utilizing immunotherapy towards achieving a functional cure for HIV-1.

PURPOSE OF REVIEW: Advancements in antiretroviral therapy (ART) have positively impacted the life expectancy and possibility of living a normal life for people with HIV-1. However, lifelong daily medication is necessary to prevent disease progression. To this end, immunotherapeutic strategies are being tested with the aim of developing a functional cure in which the immune system effectively controls HIV-1 in the absence of ART. RECENT FINDINGS: The most promising advances in achieving sustained HIV-1 remission or cure include broadly neutralizing antibodies (bNAbs) that are administered alon

Actionable Solutions to Achieve Health Equity in Chronic Liver Disease.

There are well-described racial and ethnic disparities in the burden of chronic liver diseases. Hispanic persons are at highest risk for developing nonalcoholic fatty liver disease, the fastest growing cause of liver disease. Hepatitis B disproportionately affects persons of Asian or African descent. The highest rates of hepatitis C occur in American Indian and Alaskan Native populations. In addition to disparities in disease burden, there are also marked racial and ethnic disparities in access to treatments, including liver transplantation. Disparities also exist by gender and geography, espe

Time to achieve a patient acceptable symptom state in myasthenia gravis.

INTRODUCTION: The patient acceptable symptom state (PASS) is a reliable way to characterize a patient's satisfaction with their disease state in a "Yes"/"No" dichotomous manner. There is limited data on the time required to reach an acceptable state in Myasthenia Gravis (MG). We aimed to determine the time to reach a first PASS "Yes" response in patients at MG diagnosis and a PASS "No" status, and also to determine the influence of various factors on this time. METHODS: We performed a retrospective study of patients diagnosed with myasthenia gravis who had an initial PASS "No" response and de

Importance of achieving rapid treatment response in major depressive disorder.

Major depressive disorder (MDD) is a leading contributor to disability worldwide and is associated with increased morbidity and mortality. Current pharmacologic treatment options may be ineffective for some patients and can pose several limitations and challenges, including suboptimal response and slow onset of action. Many of these therapies can take 6 to 8 weeks for patients to achieve response and 12 weeks or longer to demonstrate full clinical benefit. Delays in depressive symptom resolution are associated with poor symptomatic and functional outcomes, decreased quality of life, and increa

Introducing seasonal influenza vaccine in Bhutan: Country experience and achievements.

Bhutan successfully introduced multiple vaccines since the establishment of the Vaccine Preventable Disease Program in 1979. Surveillance and subsequent introduction of influenza vaccination became a public health priority for the Ministry of Health following the influenza A(H1N1)pdm09 pandemic. Sentinel surveillance for influenza in Bhutan began in 2008, and a study of severe acute respiratory infection was conducted in 2017, which found the highest influenza burden in children aged <5 years and adults ≥50 years. Following review of surveillance and burden of disease data, the National Techni

Can we achieve biomimetic electrospun scaffolds with gelatin alone?

Introduction: Gelatin is a natural polymer commonly used in biomedical applications in combination with other materials due to its high biocompatibility, biodegradability, and similarity to collagen, principal protein of the extracellular matrix (ECM). The aim of this study was to evaluate the suitability of gelatin as the sole material to manufacture tissue engineering scaffolds by electrospinning. Methods: Gelatin was electrospun in nine different concentrations onto a rotating collector and the resulting scaffold's mechanical properties, morphology and topography were assessed using mechani

Assessing progress towards achieving the transport dimension of the SDGs in China.

Transport is an important service industry in the national economy. Sustainable transport is central to sustainable development. Currently, investigating the sustainable development process and trade-offs in China's transport sector is urgent. In this study, 11 transport indicators were selected and constructed for the sustainable development goals (SDGs) under the UN indicator framework. The scores of each indicator were calculated, and spatiotemporal patterns and interactions were analyzed. The results revealed that China's transport infrastructure performed well in large transportation volu