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Caregiver training and support program for hospital-to-home transitions

Caregiver training and support program for hospital-to-home transitions

Unmet Need There is a need for effective caregiver training programs that reduce hospital readmissions and improve outcomes for medically vulnerable older adults. Many family caregivers are unprepared to provide complex post-discharge care at home,…

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Pediatric Hospital Medicine Infographic-Based Curriculum

Pediatric Hospital Medicine Infographic-Based Curriculum

Unmet Need There is a need for efficient, engaging, and evidence-based educational tools that can deliver key clinical concepts to pediatric trainees and practicing clinicians in time-constrained healthcare environments. Traditional didactic methods often fall short…

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Text-based cessation program for smokeless tobacco users in underserved communities

Text-based cessation program for smokeless tobacco users in underserved communities

Unmet Need There is a need for accessible, scalable, and effective smokeless tobacco cessation programs tailored to rural and medically underserved populations. Traditional interventions are often resource-intensive, web-dependent, or inaccessible due to limited internet access,…

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Remote exercise training program for older adults with rheumatoid arthritis

Remote exercise training program for older adults with rheumatoid arthritis

Unmet Need There is a need for accessible, non-pharmacologic interventions that address both cardiovascular risk and rheumatoid arthritis (RA) disease activity in older, obese adults with RA. Despite advances in pharmacologic therapies, many patients continue…

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Caregiver-centered framework for profiling caregiving experiences

Caregiver-centered framework for profiling caregiving experiences

Unmet Need There is a need for caregiver-centric frameworks that accurately capture the diversity of caregiver experiences, beyond the clinical diagnosis of the care recipient. Current models often focus on the patient’s disease state, overlooking…

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RAFT: A CRISPR-Free RNA Trans-Splicing Platform for MYH7 Correction in Hypertrophic Cardiomyopathy

RAFT: A CRISPR-Free RNA Trans-Splicing Platform for MYH7 Correction in Hypertrophic Cardiomyopathy

Unmet Need Hypertrophic cardiomyopathy (HCM) is a common inherited heart disease affecting 1 in 500 individuals, frequently caused by mutations in the MYH7 gene. Current gene therapy approaches, including AAV-based gene replacement, face significant challenges…

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Rationally designed chimeric AAV capsids for enhanced immune evasion, cellular uptake, and transduction efficiency

Rationally designed chimeric AAV capsids for enhanced immune evasion, cellular uptake, and transduction efficiency

Unmet Need Adeno-associated virus (AAV) vectors are a leading platform for in vivo gene therapy, yet their clinical potential is limited by key biological barriers. Pre-existing immunity to common AAV serotypes diminishes vector efficacy and…

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Chimeric AAV vectors enabling antibody evasion and redosing for cardiac gene therapy

Chimeric AAV vectors enabling antibody evasion and redosing for cardiac gene therapy

Unmet Need Gene therapy using adeno-associated virus (AAV) vectors is severely limited by pre-existing immunity in 30–80% of adults and by the development of neutralizing antibodies after initial treatment, preventing redosing. There is a critical…

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Methods and compositions for enhancing viral and mRNA gene therapy with engineered RNA elements

Methods and compositions for enhancing viral and mRNA gene therapy with engineered RNA elements

Unmet Need An outstanding challenge in creating effective gene therapies, particularly those based on adeno-associated virus (AAV) and mRNA delivery platforms, is overcoming sub-optimal transgene expression. Inefficiencies in translation and stability of therapeutic gene products…

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Novel AAV capsid variants for CNS-targeted gene therapies

Novel AAV capsid variants for CNS-targeted gene therapies

Unmet Need The development of effective gene therapies for central nervous system (CNS) diseases faces a critical bottleneck: current adeno-associated virus (AAV) vectors lack sufficient transduction efficiency and specificity for human brain cells. Standard AAV…

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RNA switch enabling stress-responsive regulation of protein expression for safer, more consistent gene and mRNA therapies

RNA switch enabling stress-responsive regulation of protein expression for safer, more consistent gene and mRNA therapies

Unmet Need Current gene and mRNA therapies lack mechanisms to regulate protein expression in response to intracellular stress. Overexpression of therapeutic proteins can induce ER stress leading to unfolded proteins that do more harm than…

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Muscle-targeted gene therapy for SLC25A4 mitochondrial deficiency

Muscle-targeted gene therapy for SLC25A4 mitochondrial deficiency

Unmet Need There is a significant unmet need for disease-modifying therapies that address mitochondrial dysfunction in inherited cardiomyopathies and myopathies such as SLC25A4 deficiency. Existing treatments are limited to supportive care and heart transplantation, with…

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