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Translational Paediatrics Ai notes - Coggle Diagram
Translational Paediatrics
Ai notes
Translational Research in Paediatrics
.
Importance:
Hastens the application of biomedical discoveries to patient treatment, encompassing lab studies, clinical needs, and public health.
Definition:
Translational research aims to bridge the gap between laboratory discoveries and clinical practice to improve patient care
The Need for Translational Research
Slow Translation: It can take up to 17 years for only 14% of scientific discoveries to be implemented in standard medical practice.
Examples:
Asthma: Despite evidence-based therapies, asthma rates are rising in children.
Autism and Obesity: Significant genetic, biological, and behavioral research exists, but effective human studies are needed for management.
Barriers to Translational Research
General Barriers:
Poor interaction
between bench researchers and clinical scientists.
High
cost
and limited funding.
Shrinking workforce
in clinical research and lack of training in translational methods.
Paediatric-Specific Barriers:
Small-scale
research programs with low visibility.
Diseases
are often
rare
, requiring multi-institutional collaborations that are difficult to sustain
Ongoing Projects at TCD
NIMBUS, GEMINI, FIREFLY: Focused on neonatal brain injury and perinatal pathology.
Scientific Techniques in Practice:
Use of blood samples, protein detection methods like SDS-PAGE, PCR for DNA amplification, flow cytometry, and biomarker analysis.
Success Stories in Paediatric Translational Research
Cystic Fibrosis: .
Development of KALYDECO, a drug used for cystic fibrosis patients, exemplifies translational research in action
HPV Vaccine:
The Gardasil 9 vaccine for cervical cancer prevention resulted from translational research, and Ireland provides this vaccine free to students.
Gene Therapy (Layla’s Story):
Gene therapy using UCART19 cells to treat acute lymphoblastic leukaemia shows success in genetically engineered T-cells attacking cancer.
Techniques in Translational Research
SDS PAGE: Protein detection in cell/tissue homogenates.
PCR: Amplification of DNA sequences.
Flow Cytometry: Cell counting, sorting, and detection of biomarkers.
Fluorescence Imaging: Tracks cell apoptosis and protein localization.
ATPLite Assay: Measures intracellular ATP.
Future of Paediatric Translational Research
Personalized Medicine: Tailored therapies using technologies like whole-genome sequencing and CAR T-cells for neuroblastoma treatment.
Genome Editing: CRISPR-Cas9 used to investigate OCT4’s role in human embryogenesis, paving the way for improved genetic understanding and treatments.
Advancements:
Improved Antenatal Care: Monitoring pregnancy health and using biomarkers for early diagnosis.
Government-Supported Research Centers: Collaboration across institutions to advance paediatric research.
Strengthening Approaches: Embracing new technologies, increasing funding, and training paediatric physician-scientists.
Goal: Achieving better outcomes through predictive and personalized care for children.