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Lecture 8

BIMS 201 Lecture Notes - Lecture 8: Abcd1, Demyelinating Disease, Viral Vector

Biomedical Science
Course Code
BIMS 201
Elizabeth A.Crouch

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Chapter 17A Notes
Wrongful birth bill
Problem=can viral vector travel to germ cells
Defiig hat’s seere ad ot seere
microRNAs (miRNA) = complimentary to mRNA
o shuts off protein production
o transcribed from opposite strand of DNA
o makes double-stranded RNA sequence
o 22ish base pairs long and controls production level of proteins
o anti-sense RNAs
o can be complementary to bad/good protein
hae to e looked @ so they do’t shut off good protei produtio
when genes inserted for therapy
o mRNA ad DNA are alays ade 5’ 3’
ALD: demyelinating disease of microglial cells (begin in bone marrow and travel to brain)
o Progressive brain deterioration bc demyelinating neurons = NOT transmitting
o Defect in pore protein (ABCD1) in peroxisomes
Protein makes pore in peroxisome that allows enzyme in that breaks
down long chain FAs
w/o protein: long chain FAs stay
o X-linked disease ( > boys)
Can live normal life once treated
o Successful gene therapy using HIV-stripped of everything except outer cell
(does’t hae gees that ause AIDS, has good gees for ABCD1 ad iral ode
protein that targets it to bone marrow-microglial cells)
o Newborn screen being dev. for it
o Deliver altered gene to T-helper cells (CD4+ cells) using modified HIV virus
o Can train cells to target specific cancer cells
o Leads to iue respose that’s highly speifi to your aer
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