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Gene therapy by Mind Map: Gene therapy

1. Human (blood cell disorders)

1.1. SCID

1.1.1. SCID-X1 HLA-matched bone marrow transplant retroviruses integration of provirus into host genome replication defective retro 1. CD34+ bone marrow cells isolated 2. infected with MFB(gamma)c full immune function restored in 4/5 (initial cohort) problems

2. Animal model of human

2.1. Adeno-associated virus (AAD)

2.1.1. site specific insertion

2.1.2. size limitation

2.1.3. recombination and co-transfection (helper)

2.1.4. OTC deficiency (urea cycle disorder) (But 3% of function can restore health) OTC correction in humanised mice AAV2 vector expressing mOTC intraperitoneal injection high expression

3. gene delivery vectors

3.1. liposomes

3.2. DNA transfection (gene guns, Ab conjugates)

3.3. viruses (70% of all gene therapy trials)

3.3.1. retrovirus (persistent)

3.3.2. adenovirus (transient)

3.3.3. HSV (persistent)

3.3.4. vaccinia (transient)

3.3.5. adeno-associated virus (persistent)

3.4. many human disease caused by loss of gene function

3.4.1. aim to cure by using transgenes delivered by delivery vector (complement or kill), either permanently or transiently (or even interfere using RNAi)