Breaking Boundaries in the Brain—Advances in Editing Tools for Neurogenetic Disorders Bronte A. Coorey, Wendy A. Gold
by Irem Kuyucu
1. Neurological disorders
1.1. Impaired nervous system development
1.2. Neurodegeneration
1.3. Impaired neuronal function
2. Neurogenetic disorders
2.1. Monogenic neurological disorders They are caused by a certain gene rather than multiple factors.
2.1.1. Fragile X syndrome
2.1.2. Rett syndrome
2.1.3. Huntington's disease
2.2. Inheritance
2.2.1. Recessive
2.2.2. Dominant
2.2.3. Dominant negative
2.3. Mechanism
2.3.1. Gain of function (GOF)
2.3.2. Loss of function (LOF)
2.4. Nervous system
2.4.1. Central nervous system
2.4.1.1. Seizures
2.4.1.2. Cognitive disabilities
2.4.2. Peripheral nervous system
2.4.2.1. Physical disabilities
3. Treatment
3.1. Currently available
3.1.1. No cure
3.2. Possibly available in the future
3.2.1. Gene transfer therapy
3.2.1.1. Faulty gene
3.2.1.1.1. Lack of expression
3.2.1.2. Synthetic transgenes
3.2.1.2.1. Compensation
3.2.2. Gene editing
3.2.2.1. Enzyme
3.2.2.1.1. Intentional damage
3.2.2.2. Cells
3.2.2.2.1. Natural repair mechanism
3.3. Challenges
3.3.1. Complexity of the central nervous system
3.3.2. Limited regenerative capacity of neurons
3.3.3. Blood–brain barrier
3.3.4. Dose sensitivity