Chronic obstructive pulmonary condition (COPD) is a prevalent, preventable, and curable illness with persistent respiratory symptoms and airflow limitation

2021

Preprint

Frontotemporal dementia is an uncommon and complex sickness with a wide spectrum of clinical symptoms, making drug development for the condition challenging. Frontal and temporal lobe responsibilities, as well as the level to which these activities are affected in certain diseases, contribute to clinical heterogeneity. Unfortunately, the available data on symptomatic treatments is drawn from limited case studies and RCTs, which include persons with the same FTD diagnosis. Advances in the identification of new FTD treatments demand the construction of huge clinical networks that are built on collaborative, multicenter research. One of the primary drawbacks of the present studies is the variety of the assessment instruments utilized, such as the FBI, Cambridge Behavioral Inventory, NPI, and BEHAVE-AD, some of which were not originally established for FTD but were first validated for other kinds of dementia, such as AD. The FBI was shown to have high sensitivity and specificity in discriminating between bvFTD and non-FTD, whereas the NPI score did not.The new definition of Alzheimer's disease was changed from a clinical diagnostic to a biomarker-based diagnostic, although we are still a long way from providing a scientific description of FTD. In the future, there might be an increased likelihood of recognizing symptoms or signs in clinical studies because of a revision to the criteria of FTD.Treatment techniques should be customized to individual patient subgroups or mutation carriers. Currently, as a treatment for FTD-tau, antisense oligonucleotide inhibition of MAPT, tau phosphorylation inhibition, microtubule stability, and inhibition of tau aggregation are all being investigated. It is possible to inhibit TDP-43 aggregation, enhance progranulin levels, activate the autophagy–lysosome system, or modify the ubiquitin-proteasome system in FTD-TDP. Despite these breakthroughs, the etiology and genetics of FTD remain unclear. Additional genetic pathways and loci associated with immune dysregulation and inflammation have recently been discovered, furthering the search for possible treatment targets. Also, knowing who the mutation carriers are early on allows researchers to test disease-modifying drugs earlier, when cognitive problems may still be reversible.Research gaps in the etiology and clinical characterization of FTD should be resolved, as well as the production of precise biomarkers and specialized evaluation methodologies. Desharnais and colleagues have created a checklist to assist standardize future FTD clinical trials and boost the probability of successful findings.

“…Lithium was poorly tolerated and linked to an increased risk of falls, drowsiness, and tremor, particularly at high dosages (about 1200 mg/day) (53).…”

Section: Current Treatments For Ftd Tolerance and Safetymentioning

confidence: 99%

Frontotemporal dementia is a complex disorder with a wide spectrum of clinical symptoms. Personalized medicine and gene therapy are promising strategies for treatment

2021

Preprint

“…Vitamin D has also been shown to be effective (28,56). Anti-inflammatory drugs, such as anti-interleukin12/23 monoclonal antibody, anti-tumor necrosis factor agents, and enzyme replacement therapy, such as topical recombinant human TGm-1 liposomes, recombinant kallikrein, and-HAC, have been shown to be effective in cell lines and animal models, and warrant further research (28,(57)(58)(59)(60). Ex vivo gene editing also improved filament integrity in EI keratinocytes (61).…”

Section: Treatment and Diagnosismentioning

confidence: 99%

Gene therapy may benefit inherited ichthyoses with concurrent fungal infections and severe ich thyroidoses

2021

Preprint

Despite the fact that inherited ichthyoses with simultaneous fungal infections have been documented for more than 50 years, the connection between the two remains unknown. Because ichthyosis makes it harder to identify a concurrent fungal infection, it's important to pay close attention to prevent missing or delaying a diagnosis. The underlying ichthyosis makes treating fungal infections more difficult, and severe and recurrent infections need further attention. Those with severe ichthyoses may benefit from gene therapy in order to avoid fungal infection. More clinical and laboratory research is needed, however, to fully comprehend the mechanisms of action and provide a foundation of knowledge from which innovative therapy alternatives might be created.

“…Furthermore, current influenza vaccines have a short shelf life, with antibody titers dropping within a year (59), needing yearly reformulation and booster immunization. STING agonists have been used to increase vaccine protection in continuing efforts to develop a broadly neutralizing influenza vaccine (60,61).…”

Section: Hanson Et Al Demonstrated Two Liposome-based Hiv Vaccines Wi...mentioning

confidence: 99%

Stimulator of interferon genes (STING)-activating nanoparticles can be employed as a tool for controlled immune activation

2021

Preprint

Advances in materials and chemistry have helped stimulator of interferon genes (STING) agonists deliver nanomedicine, and drug delivery scientists have generated a spate of STING-activating nanocarriers capable of bypassing biological barriers for targeted innate and adaptive immune activation. Using STING-activating nanoparticles in antiviral vaccination has yielded several promising results in preclinical studies, and the strong antiviral humoral and cellular immunity elicited by these particulate adjuvants against HIV, influenza, and coronaviruses justifies the continued development of the platforms against the global infectious threat. STING-activating nanoparticles can be employed in virology and immunology research as a tool for controlled immune activation, as many processes underpinning STING-mediated immunological potency in viral infections are yet unclear. Because many of the nanoparticle platforms used to deliver STING agonists may also be utilized with various molecular adjuvants, these systems may be utilized to examine how diverse innate immune signals impact adaptive immunity production. Combinatorial nanoparticles mixing STING agonists with other molecular adjuvants may further increase antiviral protectivity by synergistically increasing immunological potential. Despite the promise of nanoparticulate vaccines, these synthetic nanoparticles are strikingly absent from COVID-19 vaccine research worldwide talks. Many elements, such as formulation safety, manufacturing scalability, storage, and deployment logistics, must be considered to achieve the enormous potential of the nanoparticulate STING agonists. Reduced synthetic methods and formulation lyophilization are important factors in platform designs to make antiviral vaccines accessible and affordable to poor nations. Nevertheless, the platform's great efficiency and wide use provide promise advances in both fundamental and translational research, opening the way to a deeper knowledge of viral containment and superior approaches.