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The potential treatment strategies for mitochondrial diseases (Diseases…
The potential treatment strategies for mitochondrial diseases
Introduction
Statistics of people suffering from mitochondrial diseases
Thesis statement
Diseases caused by mitochondrial disorders
Current treatments provided by healthcare services
General information of mitochondria
Clinical trails and approach
Discussion
Conclusion
Causes of mitochondrial diseases
Available treatment for mitochondrion diseases
Supportive, relieving the symptoms that can be developed
Make energy production more efficient
By using co-factors and vitamins
By using special diet controlled by metabolic team/professionals
General Information of mitochondria
Origin
37 chromosomes
16,000 base pairs
Hypothesis
Mitochondria was engulfed by eukaryotic cells via phagocytosis
Mitochondria were a metabolically versatile, being commensalism with a prokaryotic cell. However, having similarities in physiology and lifestyle, these two organisms had developed to live together.
Mitochondrial inheritance
The inheritance of a trait encoded in mitochondria chromosome
In sexual production, mitochondria are exclusively inherited from mother, since the zygote is developed from the egg cell from the mother.
Structures
Inner membrane
Outer membrane
Cristae
Produce Energy
Matrix
Ribosomes
Roles and functions
Production of energy in the term of Adenosine Triphosphate (ATP) via Citric Acid cycle
Production of heat (Non-shivering thermogenesis)
Programmed cell death (Apoptosis)
Storage of Calcium (2+) ions
Current treatments
Pre-clinical development
Gene therapy
Stem cell therapy
Diseases caused by mitochondria
Alper's Disease
Mitochondrial DNA Depletion Syndrome (MDDS)
Leigh Syndrome
NARP - Neurogenic Weakness, Ataxia and Retinitis Pigmentosa
Leber's Hereditory Optic Neuropathy (LHON)
Causes of mitochondrial diseases
MELAS - Mitochondrial Encephalomyopathy, Lactic Acidosis and stroke like episodes 3243A>G Mutation
MNGIE - Mitochondrial Neuro-Gastro-Intestinal Encephalopathy
Mitochondrial DNA deletion
Multiple Mitochondrial DNA Deletions
Refers to different sized pieces of mitochondrial DNA that are missing.
It is not due to a genetic fault (mutation) in mitochondrial DNA.
Large Scale mitochondrial DNA Deletions - Kearn's-Sayre Syndrome and Pearson's Syndrome
Describes a piece of DNA that is missing from lots of copies of mitochondrial DNA in each cell
It occurs by chance (sporadic)
Clinical trails and approach
Cas9
Mitochondrial gene sequences
Figure out the mitochondrial disorder leading to the understanding about the disease
Discussion
Advantages
Disadvantages
Risks
Cost and benefitients
Places (which the treatment is available)
Conclusion
The most potential strategy for mitochondrion diseases
Reasons
Suggestions
