Patient Centricity Driving Formulation Innovation: Improvements in Patient Care Facilitated by Novel Therapeutics and Drug Delivery Technologies

2022 ◽  
Vol 62 (1) ◽  
pp. 341-363
Author(s):  
Susanne Page ◽  
Tarik Khan ◽  
Peter Kühl ◽  
Gregoire Schwach ◽  
Kirsten Storch ◽  
...  
Keyword(s):  
Patient Care ◽  
Targeted Delivery ◽  
Messenger Rna ◽  
Medical Information ◽  
Digital Platforms ◽  
Therapeutic Modalities ◽  
Age Appropriate ◽  
Patient Groups ◽  
Sites Of Action ◽  
Interfering Rna

Innovative formulation technologies can play a crucial role in transforming a novel molecule to a medicine that significantly enhances patients’ lives. Improved mechanistic understanding of diseases has inspired researchers to expand the druggable space using new therapeutic modalities such as interfering RNA, protein degraders, and novel formats of monoclonal antibodies. Sophisticated formulation strategies are needed to deliver the drugs to their sites of action and to achieve patient centricity, exemplified by messenger RNA vaccines and oral peptides. Moreover, access to medical information via digital platforms has resulted in better-informed patient groups that are requesting consideration of their needs during drug development. This request is consistent with health authority efforts to upgrade their regulations to advance age-appropriate product development for patients. This review describes formulation innovations contributingto improvements in patient care: convenience of administration, preferred route of administration, reducing dosing burden, and achieving targeted delivery of new modalities.

scholarly journals Redox Responsive Pluronic Micelle Mediated Delivery of Functional siRNA: A Modular Nano-Assembly for Targeted Delivery

2021 ◽  
Author(s):  
Sandeep Kadekar ◽  
Ganesh N. Nawale ◽  
Vignesh Kumar Rangasami ◽  
Vadim Le Joncour ◽  
Pirjo Laakkonen ◽  
...  
Keyword(s):  
Targeted Delivery ◽  
Unmet Need ◽  
Short Interfering Rna ◽  
Site Specific ◽  
Redox Responsive ◽  
Interfering Rna

There is an unmet need to develop strategies that allow site-specific delivery of short interfering RNA (siRNA) without any associated toxicity. To address this challenge, we have developed a novel...

Staffing Patterns for Patient Care and Support Personnel in a General Pediatric Unit

PEDIATRICS ◽  
1994 ◽  
Vol 93 (5) ◽  
pp. 850-854 ◽  
Author(s):  
Keyword(s):  
Patient Care ◽  
Public Hospital ◽  
Inpatient Unit ◽  
Her Family ◽  
Appropriate Care ◽  
Care And Support ◽  
Staffing Patterns ◽  
Age Appropriate ◽  
Pediatric Inpatient ◽  
Care Outcomes

The mission of a pediatric inpatient unit, no matter how large or small, whether in a private or public hospital, is to provide optimum, age-appropriate care for each patient and to lend sensitive and understanding support to his or her family. The key to success in achieving this mission is the quality and commitment of the personnel on the unit. If the skills and the dedication are present and there is flexibility in staffing assignments, the desired patient care outcomes will be inevitable.

scholarly journals Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy

Molecules ◽  
2020 ◽  
Vol 25 (12) ◽  
pp. 2866 ◽  
Author(s):  
Aniket Wahane ◽  
Akaash Waghmode ◽  
Alexander Kapphahn ◽  
Karishma Dhuri ◽  
Anisha Gupta ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Messenger Rna ◽  
Viral Vectors ◽  
Cationic Lipids ◽  
Nucleic Acid Delivery ◽  
Nucleic Acid Analogs ◽  
Organ Specific ◽  
Interfering Rna

The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been developed to target coding or non-coding sequences of the human genome for gene regulation. However, broader clinical applications of nucleic acid analogs have been limited due to their poor cell or organ-specific delivery. To resolve these issues, non-viral vectors based on nanoparticles, liposomes, and polyplexes have been developed to date. This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications.

Children’s views on taking medicines and participating in clinical trials

2019 ◽  
Vol 104 (9) ◽  
pp. 900-905 ◽  
Author(s):  
Sofia Nordenmalm ◽  
Elin Kimland ◽  
Franca Ligas ◽  
Birka Lehmann ◽  
Joana Claverol ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Medical Information ◽  
Chronic Health Condition ◽  
Health Condition ◽  
Limited Information ◽  
European Union Countries ◽  
Age Appropriate ◽  
Common Problems ◽  
Children’S Views

IntroductionLimited information is available on the views of children taking medicines and participating in clinical trials. These views may contribute to a better understanding of what can be improved on in the development of medicines from their perspective.ObjectiveTo collect children’s views on taking medicines and participating in clinical trials.Materials and methodsA question-based survey was conducted among children living in European Union countries between January and August 2015.ResultsAlmost 900 children aged 10–17 years from Finland, Germany, Sweden, Spain and Hungary responded. Almost 40% had a chronic health condition. The most commonly used pharmaceutical forms were solid or liquid medicines for oral use and injectable medicines. Bad taste and pain during administration were reported as common problems. Of 785 respondents, 17% had been taking part in a clinical trial. Most respondents would potentially agree to take part in a clinical trial because the investigational medicine might improve their own health or that of other children. Concern that the investigational medicine might be harmful was the main reason to refuse participation, if asked to. Over half of the respondents were willing to learn more about clinical trials, preferably online.ConclusionsIt is necessary to involve children in the development of age-appropriate pharmaceutical forms and in the design of clinical trials. Children and their carers should be provided with age-appropriate medical information in the most suitable channels. We have identified some common problems that children experience when taking medicines, and we conclude that children are interested in learning more and giving their opinions on clinical trials.

Design of cyclic RGD-conjugated Aib-containing amphipathic helical peptides for targeted delivery of small interfering RNA

2016 ◽  
Vol 24 (18) ◽  
pp. 4478-4485 ◽  
Author(s):  
Shun-ichi Wada ◽  
Masashi Iwata ◽  
Yuka Ozaki ◽  
Takashi Ozaki ◽  
Junsuke Hayashi ◽  
...  
Keyword(s):  
Targeted Delivery ◽  
Small Interfering Rna ◽  
Helical Peptides ◽  
Interfering Rna

scholarly journals A conserved CCCH-type zinc finger protein regulates mRNA nuclear adenylation and export

2009 ◽  
Vol 185 (2) ◽  
pp. 265-277 ◽  
Author(s):  
Jessica A. Hurt ◽  
Robert A. Obar ◽  
Bo Zhai ◽  
Natalie G. Farny ◽  
Steven P. Gygi ◽  
...  
Keyword(s):  
Zinc Finger ◽  
Nuclear Export ◽  
Messenger Rna ◽  
Zinc Finger Protein ◽  
Liquid Chromatography Mass Spectrometry ◽  
Functional Conservation ◽  
Finger Protein ◽  
Interfering Rna ◽  
Processing Steps ◽  
Prior Processing

Coupling of messenger RNA (mRNA) nuclear export with prior processing steps aids in the fidelity and efficiency of mRNA transport to the cytoplasm. In this study, we show that the processes of export and polyadenylation are coupled via the Drosophila melanogaster CCCH-type zinc finger protein CG6694/dZC3H3 through both physical and functional interactions. We show that depletion of dZC3H3 from S2R+ cells results in transcript hyperadenylation. Using targeted coimmunoprecipitation and liquid chromatography mass spectrometry (MS)/MS techniques, we characterize interactions of known components of the mRNA nuclear export and polyadenylation machineries with dZC3H3. Furthermore, we demonstrate the functional conservation of this factor, as depletion of its human homologue ZC3H3 by small interfering RNA results in an mRNA export defect in human cells as well. Nuclear polyadenylated (poly(A)) RNA in ZC3H3-depleted cells is sequestered in foci removed from SC35-containing speckles, indicating a shift from the normal subnuclear distribution of poly(A) RNA. Our data suggest a model wherein ZC3H3 interfaces between the polyadenylation machinery, newly poly(A) mRNAs, and factors for transcript export.

Pain medicine

2014 ◽  
Author(s):  
Jeremy Prout ◽  
Tanya Jones ◽  
Daniel Martin
Keyword(s):  
Chronic Pain ◽  
Pain Management ◽  
Management Strategies ◽  
Multidisciplinary Management ◽  
Pain Medicine ◽  
Pain Pathways ◽  
Patient Groups ◽  
Chronic Pain Conditions ◽  
Sites Of Action ◽  
Specific Management

This chapter summarizes the assessment and management of acute and chronic pain for FRCA. Pain pathways and physiological consequences of pain are considered along with sites of action and the pharmacology of common analgesics. Assessment of pain for different patient groups and settings is explained. Pain management strategies, pharmacological, non-interventional and interventional techniques are described, including multidisciplinary management of chronic pain. Specific management of some common chronic pain conditions, such as trigeminal neuralgia, are discussed in more detail.

scholarly journals Inhibition of PTEN Gene Expression by Small Interfering RNA on PI3K/Akt/FoxO3a Signaling Pathway in Human Nasopharyngeal Carcinoma

2020 ◽  
Vol 19 ◽  
pp. 153303382091795
Author(s):  
Liang Zhong Yao ◽  
Yan Li Zhu ◽  
Jun Jie Liu
Keyword(s):  
Nasopharyngeal Carcinoma ◽  
Signaling Pathway ◽  
Messenger Rna ◽  
Small Interfering Rna ◽  
Quantitative Polymerase Chain Reaction ◽  
Pten Gene ◽  
Control Group ◽  
Expression Levels ◽  
Human Nasopharyngeal Carcinoma ◽  
Interfering Rna

The objective of this article is to study the effect of inhibiting phosphatase and tensin homolog deleted chromatosome 10 gene on phosphoinositide 3-kinase/protein kinase B ( Akt)/Forkhead homeobox O3a signaling pathway in human nasopharyngeal carcinoma HK-1 cells. Nasopharyngeal carcinoma HK-1 cell lines were divided into PTEN gene interference group (siPTEN), nonspecific small interfering RNA group (siNC), empty vector group (Vector), and no transfection control group (Normal). The mRNA and protein expression levels of PTEN, PI3K, p-Akt, and FoxO3a were detected by real-time fluorescence quantitative polymerase chain reaction and Western blot. Immunofluorescence was used to detect the subcellular localization of PTEN, PI3K, p-Akt, and FoxO3a in HK-1 cells. The proliferation of HK-1 cells was detected by MTT assay, and the apoptosis of HK-1 cells was detected by flow cytometry. Compared with the siNC group, the expression levels of PTEN, FoxO3a messenger RNA, and protein in the siPTEN group were significantly decreased ( P < .05), while the expression levels of PI3K, p-Akt messenger RNA, and protein were significantly increased ( P < .05). The growth rate of HK-1 cells in the siPTEN group was significantly higher than the siNC group ( P < .05), while the apoptosis rate was significantly lower than that of the siNC group ( P < .05). Small interfering RNA can inhibit the expression of PTEN in HK-1 cells, and PTEN can participate in the development of NPC by affecting PI3K/Akt/FoxO3a signaling pathway.

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