GENE THERAPY FOR HAEMOPHILIA

The blood disorder, Hemophilia, has its roots embedded deep into the history of genetic disorders. The European royal family is one of the most prominent families to be affected by this disease thus, dubbing it 'the royal disease'. The types of Hemophilia are divided into two based on the type of coagulation factor mutation found in the patient. For treating haemophilia, gene therapy is done by using different vectors such as lentiviral and retroviral vectors but due to the production of limited expression different adeno associated virus (AAV) strains are used. Some engineerly modified vectors are currently used to get the best possible results. The clinical trials prove the efficacy of these vectors so through their obtained statistical consideration, patient experience and population study once can design vaccines and drugs for haemophilia patients but also due to pre-existing Nabs and pre-existing HCV or HBV infection, the general application of AAV gene therapy is currently limited. The possibility of gene editing for the repair of the mutation is on the horizon.

Statistical consideration when adding new arms to ongoing clinical trials: the potentials and the caveats

Background Platform trials improve the efficiency of the drug development process through flexible features such as adding and dropping arms as evidence emerges. The benefits and practical challenges of implementing novel trial designs have been discussed widely in the literature, yet less consideration has been given to the statistical implications of adding arms. Main We explain different statistical considerations that arise from allowing new research interventions to be added in for ongoing studies. We present recent methodology development on addressing these issues and illustrate design and analysis approaches that might be enhanced to provide robust inference from platform trials. We also discuss the implication of changing the control arm, how patient eligibility for different arms may complicate the trial design and analysis, and how operational bias may arise when revealing some results of the trials. Lastly, we comment on the appropriateness and the application of platform trials in phase II and phase III settings, as well as publicly versus industry-funded trials. Conclusion Platform trials provide great opportunities for improving the efficiency of evaluating interventions. Although several statistical issues are present, there are a range of methods available that allow robust and efficient design and analysis of these trials.

Some statistical consideration of azimuth and inclination angles determination based on walk - away VSP data in Python

It is a common knowledge that proper inclinations and azimuth angle determination is a critical step in processing and interpretation of walk-away VSP data. Additionally, an in-depth analysis of the uncertainty of these interpreted values requires the introduction of measurement errors. In this contribution, we present a statistical analysis of obtained polarization angles from three-component, multi-depth level, walk-away VSP using Python 3 programming language. Our analysis is presented in the context of different processing sequences and correlation with local features of the geological medium. We show that the obtained values of polarization angles and their errors can be strongly affected by processing sequence and - when done correctly - can give addition inside into features of analysis medium. Moreover, in some cases, even a presence of saturation can be express by polarization angles variations. Additionally, we examined the impact of well-casing on interpretational values of polarization angles.

On the Role of Accent in Ancient Greek Poetry

In this paper, I set out to study the distribution of some accentual types of two different word shapes in the Homeric hexameter: iambs and iambic ending words and dactyls and dactylic ending words. Following Nagy 2000 and 2010 and David 2006, I start from the idea that accent has a role in Ancient Greek poetry, which has been corroborated in Abritta 2015 by studying the distribution of trochaic ending words. In the sections corresponding to each word shape, after a statistical consideration of the distribution, I examine some literary uses of accentual types, which the poet places in the line apparently in order to produce a certain effect on the audience. The article is conceived as a first approximation to the study of accentual distribution in Ancient Greek poetry.

Statistical Consideration of Grain Growth Mechanism of Multicrystalline Si by One-Directional Solidification Technique

The grain evolution of multicrystalline Si was studied using the ingot grown from microcrystalline template. The grain shape evolution and width increase are not monotonic but may have 3 stages. On the other hand, the grain boundary (GB) analysis suggests that there exit 2 reactions, namely random GB annihilation at the initial stage and Σ3 generation and annihilation at the steady state.