Paediatric research sets new standards for therapy in paediatric and adult cholestasis.

Children with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC) experience debilitating pruritus, for which there have been few effective treatment options. In the past 2 years, the ileal bile acid transporter (IBAT) inhibitors maralixibat and odevixibat have been approved for the management of cholestatic pruritus in these individuals, representing an important step forward in improving their quality of life. Emerging data suggest these drugs might also improve event-free survival, therefore potentially altering the typical disease course currently seen in these disorders.

ATP7B Genotype and Chronic Liver Disease Treatment Outcomes in Wilson Disease: Worse Survival With Loss-of-Function Variants.

Background And Aims: Although a good genotype-phenotype correlation has not been established in Wilson disease (WD), patients with loss-of-function (LOF) ATP7B variants demonstrate different clinical and biochemical characteristics. We aim to describe long-term treatment outcomes in the chronic liver disease (CLD) phenotype and evaluate an association with LOF variants.

Methods: This was a single-center retrospective review of WD patients with at least 1 variant in ATP7B.

Nasobiliary drainage: an effective treatment for pruritus in cholestatic liver disease.

Introduction: Nasobiliary drains (NBDs) have been successfully used to manage intrahepatic cholestasis, bile leaks and obstructive cholangitis. It allows external drainage of bile, bypassing the ileum where bile salts are reabsorbed. We assessed the utility of placement with effect on markers of cholestasis and patient symptoms.

Rare Inherited Cholestatic Disorders and Molecular Links to Hepatocarcinogenesis.

Hepatocellular carcinoma (HCC) is the most common primary liver cancer affecting adults and the second most common primary liver cancer affecting children. Recent years have seen a significant increase in our understanding of the molecular changes associated with HCC. However, HCC is a complex disease, and its molecular pathogenesis, which likely varies by aetiology, remains to be fully elucidated.

Outcomes in Clinical Trials Evaluating Interventions for the Prevention and Treatment of Hepatic Encephalopathy.

Randomised clinical trials and systematic reviews of research findings can provide high-quality evidence for decision-making in the management of patients with hepatic encephalopathy. A large number of clinical trials have been undertaken, over the last 50 years, relative to the prevention and treatment of this condition. However, changes have been made, during this time, in the classification of hepatic encephalopathy, diagnostic criteria and assessment measures.

Carvedilol versus traditional, non-selective beta-blockers for adults with cirrhosis and gastroesophageal varices.

Background: Non-selective beta-blockers are recommended for the prevention of bleeding in people with cirrhosis, portal hypertension and gastroesophageal varices. Carvedilol is a non-selective beta-blocker with additional intrinsic alpha-blocking effects, which may be superior to traditional, non-selective beta-blockers in reducing portal pressure and, therefore, in reducing the risk of upper gastrointestinal bleeding.

Objectives: To assess the beneficial and harmful effects of carvedilol compared with traditional, non-selective beta-blockers for adults with cirrhosis and gastroesophageal varices.

Nanotechnology in neurosurgery: thinking small, dreaming big.

Nanotechnology has the power to transform neurosurgery by facilitating intervention at the cellular and subcellular level. The unique properties of nanomaterials will not only improve the management of conditions traditionally treated through neurosurgery, but also make neurosurgical intervention possible for diseases where there are currently limited treatment options. Specifically, nanotechnology appears to be a promising tool for improving molecular imaging, seamlessly integrating diagnosis and therapy in neuro-oncology, identifying targets for selective neuromodulation, as well as promoting neuroregeneration.

Pharmaceuticals and Stem Cells in Autism Spectrum Disorders: Wishful Thinking?

Objective: We provide a contemporary account of the key pathologic events pertaining to autism: the theory of oxidative stress and inflammatory causes, ideas of immune dysfunction, the probable biomarkers that can be used for diagnostics, and the use of pharmaceuticals and stem cells as possible candidates for the treatment of autism spectrum disorders (ASDs).

Methods: ASDs are a group of complex neurodevelopmental conditions characterized by abnormal patterns of attention and impaired social and communication skills. ASDs are also associated with numerous functional challenges and potentially harmful deficits, including restricted and repetitive behaviors, anxiety, irritability, seizures, and self-harm.

Self-assembly and sequence length dependence on nanofibrils of polyglutamine peptides.

Huntington's disease (HD) is recognized as a currently incurable, inherited neurodegenerative disorder caused by the accumulation of misfolded polyglutamine (polyQ) peptide aggregates in neuronal cells. Yet, the mechanism by which newly formed polyQ chains interact and assemble into toxic oligomeric structures remains a critical, unresolved issue. In order to shed further light on the matter, our group elected to investigate the folding of polyQ peptides - examining glutamine repeat lengths ranging from 3 to 44 residues.

Nanotechnology and regenerative therapeutics in plastic surgery: The next frontier.

The rapid ascent of nanotechnology and regenerative therapeutics as applied to medicine and surgery has seen an exponential rise in the scale of research generated in this field. This is evidenced not only by the sheer volume of papers dedicated to nanotechnology but also in a large number of new journals dedicated to nanotechnology and regenerative therapeutics specifically to medicine and surgery. Aspects of nanotechnology that have already brought benefits to these areas include advanced drug delivery platforms, molecular imaging and materials engineering for surgical implants.

Vascularisation in regenerative therapeutics and surgery.

Vascularisation is often deemed the holy grail of tissue engineering because it is one of the key preconditions that determine the in vivo viability of tissue constructs. Given that a well-developed vascular network allows greater complexity in tissue design and helps regulate tissue metabolism, it appears that the overall outcome of engineered tissue implants depends on the success of microvessel formation, maturation and patterning. Current approaches to vascularising tissue include both in vivo and ex vivo techniques, where blood vessel formation is either spontaneous or guided by physical or biochemical factors.