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1.
J Gene Med ; 26(2): e3672, 2024 Feb.
Article in English | MEDLINE | ID: mdl-38380796

ABSTRACT

Ribonucleic acid (RNA) has emerged as one of the most promising therapeutic payloads in the field of gene therapy. There are many unique types of RNA that allow for a range of applications including vaccination, protein replacement therapy, autoimmune disease treatment, gene knockdown and gene editing. However, RNA triggers the host immune system, is vulnerable to degradation and has a low proclivity to enter cells spontaneously. Therefore, a delivery vehicle is required to facilitate the protection and uptake of RNA therapeutics into the desired host cells. Lipid nanoparticles have emerged as one of the only clinically approved vehicles for genetic payloads, including in the COVID-19 messenger RNA vaccines. While lipid nanoparticles have distinct advantages, they also have drawbacks, including strong immune stimulation, complex manufacturing and formulation heterogeneity. In contrast, synthetic polymers are a widely studied group of gene delivery vehicles and boast distinct advantages, including biocompatibility, tunability, inexpensiveness, simple formulation and ease of modification. Some classes of polymers enhance efficient transfection efficiency, and lead to lower stimulation of the host immune system, making them more viable candidates for non-vaccine-related applications of RNA medicines. This review aims to identify the most promising classes of synthetic polymers, summarize recent research aimed at moving them into the clinic and postulate the future steps required for unlocking their full potential.


Subject(s)
Nanoparticles , RNA , Polymers , Gene Transfer Techniques , Transfection , Genetic Therapy
2.
Physiother Theory Pract ; 31(1): 29-38, 2015 Jan.
Article in English | MEDLINE | ID: mdl-24983454

ABSTRACT

PURPOSE: To evaluate a program in support of chronic disease self-management (CDSM) that is founded on a health coaching (HC) approach, includes supervised exercise and mindfulness-based stress reduction components and is delivered within a private practice physiotherapy setting. METHODS: An explanatory mixed method design, framed by theory-based program evaluation, was employed to evaluate an eight-week group-based program. Standardized self-rated and performance measures were evaluated pre- and post intervention. Additionally, participant focus groups were conducted following the intervention period. An inductive thematic approach was undertaken to analyze the qualitative data. FINDINGS: Seventeen participants (N = 17) completed the study. Improvements were seen in both self-report and performance outcomes. Participants explained how and why they felt the program was beneficial. Six themes were generated: (1) group dynamic; (2) learning versus doing; (3) holism and comprehensive care; (4) self-efficacy and empowerment; (5) previous solutions versus new management strategies; and (6) healthcare provider support. CONCLUSIONS: This study established that a group program in support of CDSM founded on a HC approach demonstrated potential value from participants as well as favorable outcomes. A pragmatic randomized control trial is required to determine efficacy of this intervention.


Subject(s)
Chronic Disease/rehabilitation , Motor Activity/physiology , Patient Education as Topic/organization & administration , Physical Therapists/organization & administration , Self Care/methods , Aged , Evaluation Studies as Topic , Female , Focus Groups , Humans , Long-Term Care , Male , Middle Aged , Professional-Patient Relations , Program Evaluation , Psychotherapy, Group/organization & administration , Quality of Life , Surveys and Questionnaires , Treatment Outcome
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