Drug Name

Type

Azidothymidine (AZT)

thymidine analog

gets phoshorylated to
nucleoside; lacks 3' OH

Didanosine

adenosine analog

gets phoshorylated to
nucleoside; lacks 3' OH
open chain struc instead of
pentose ring, causing

Acyclovir

guanine analog

termination of DNA
rpln; converted to acyclovir
triphosphate by thymidine
kinase

Tenofovir

AMP analog

lacks pentose ring

cytosine arabinoside "Cytarabine"

deoxycytidine analog

unusual planar struc

induces DNA damage

adenosine arabinoside "Vidarabine"

deoxyadenosine analog

unusual planar struc

induces DNA damage

5-aza-2'-deoxycytidine "Decitabine"

cytidine analog

disrupts epigenetic process

of methylation; has N atom
at 5' position of cytidine

5-azacytidine

cytidine analog

disrupts epigenetic process

of methylation; has N atom
at 5' position of cytidine

5-fluorouracil - THYMINE

uracil analog

converted to FdUMP;
inhibits syn of thymine
nucleotides

azathioprine - ORGAN

purine analog

metabolized into 6mercaptopurine (a purine syn

inhibitor)

allopurinol - GOUT

purine analog

lowers conversion of purines

to uric acid end products
(xanthine, hypoxanthine)

adenosine 4 - SLOW HEART

purine (nucleoside)

slows heart rate

ciprofloxacin

quinolone

inhibits gyrase activity

nalidixic acid

quinolone

novobiocin

coumarin

camptothecin

n/a

etoposide

n/a

prevents topoisomerase II
from re-ligating DNA;
apoptosis

actinomycin D

Streptomyces strain, fungus

binds tightly to dsDNA

build-up of positive supercoils

around rpln fork
n/a
prevents topoisomerase I
from re-ligating DNA; DNA
breakage

rifampin

Streptomyces strain, fungus

blocks formation of 1st

phosphodiester bond in txn
initiation

alpha-amanitin MUSHROOM

toxin

blocks mRNA production

diptheria toxin

bacterial exotoxin

inactivates EF-2;
disrupts translocation of
mRNA

streptomycin - 30

Streptomyces strain;
aminoglycoside

prevents ribosome assembly

by binding to 30S

tetracycline - ELONGATION
70

4-ring struc; Streptomyces

strain

binds 70S ribosome to

prevent access to aminoacyltRNA

erythromycin TRANSLOCATION - 70
chloramphenicol - PEPTIDYL
TRANSFERASE
cycloheximide - PEPTIDYL
TRANSFERASE

macrolide struc.

binds to 70S ribosome

n/a

n/a

n/a

blocks peptidyl transferase

puromycin

aminoacyl-tRNA structural
analog

causes peptide to fall out of

ribosomal P site

Rotenone

Complex I of ETC

Piericidin A

Complex I of ETC

Amytal

Complex I of ETC

Antimycin A

Complex III of ETC

CO

Complex IV of ETC

Inhibit Cytochrome Oxidase

Azide

Complex IV of ETC

Inhibit Cytochrome Oxidase

Hydrogen Sulphide

Complex IV of ETC

Inhibit Cytochrome Oxidase

Cyanide

Complex IV of ETC

Inhibit Cytochrome Oxidase

Oligomycin

Complex V of ETC

Inhibits ATP Synthase

Atractyloside

Binds outer portion

(innermembrane space) of
Adenine Nucleotide
Transporter

Inhibits ADP/ATP transport

Bongkrekic Acid

Binds inner portion (matrix) of

Adenine Nucleotide
Transporter

Inhibits ADP/ATP transport

Inhibitit NADH
Dehydrogenase
Inhibitit NADH
Dehydrogenase
Inhibitit NADH
Dehydrogenase
Inhibits cytochrome B of
Cytochrome Reductase

DNP

Uncoupler of ox phos

Destroys Proton Gradient

Aspirin

Uncoupler of ox phos

Destroys Proton Gradient

Thermogenin (UCP)

Uncoupler of ox phos

Destroys Proton Gradient

Valinomycin (ionophore)

Mobile Carrier of K+ (polar

inside, nonpolar outside)

Destroys Proton Gradient

Gramicidin (ionophore)

forms channel within

membrane

Destroys Proton Gradient

Targets prok/euk?

Inhibited Enzyme

Inhibited Cell process

prok

reverse transcriptase, RNAdependent DNApol, viral

DNA rpln

prok

reverse transcriptase, RNAdependent DNApol, viral

DNA rpln

prok

DNApol, viral

DNA rpln

prok

reverse transcriptase, RNAdependent DNApol, viral

DNA rpln

euk

DNApol

DNA rpln

euk

DNApol

DNA rpln

euk

DNA methyltransferase

DNA methylation, gene

expression

euk

DNA methyltransferase

DNA methylation, gene

expression

euk

thymidylate kinase

DNA rpln

euk

purine syn enzymes

DNA rpln

euk

xanthine oxidase

purine synthesis

euk

n/a

n/a

prok

DNA gyrase, bac

DNA txn, rpln

prok

DNA gyrase, bac

DNA rpln

prok

DNA gyrase, bac

DNA txn, rpln

euk

topoisomerase I

DNA txn, rpln

euk

topoisomerase II

DNA rpln

prok + euk

RNApol

DNA txn, rpln

prok

RNApol

DNA txn

euk

RNApol II

DNA txn

euk

GTPase

RNA tln, mRNA translocation

prok

n/a

RNA tln, initiation

prok

70S ribosome

RNA tln, elongation

prok

70S ribosome

RNA tln, translocation

prok

peptidyl transferase

RNA tln, elongation

euk

peptidyl transferase

RNA tln, elongation

prok + euk

aminoacyl-tRNA

RNA tln, elongation

insecticide
bacterial antibiotic

antibiotic

is a streptomyces antibiotic
toxic glycoside from a plant has sugar and noncarb
element
respiratory toxin - produced in
coconuts

protein in the mito of brown

adipose tissue in kids;
dissipation of H+ gradient
that doesn't go through ATP
synthase, generates heat

activation of thermogenin due

to hormone induced release
of fatty acids from
triglycerides in the brown
tissue

make inner membrane

permeable to compounds that
cant usually cross - increases
proton permeability

is a streptomyces antibiotic

make inner membrane

permeable to compounds that
cant usually cross - increases
proton permeability

Applications

Notes

targets retrovirus replication

(i.e. HIV)

100-300x affinity for reverse

transcriptase over DNApol;
minimal effect on DNApol

targets retrovirus replication

(i.e. HIV)

100-300x affinity for reverse

transcriptase over DNApol;
minimal effect on DNApol

herpes (HSV) treatment

treat HIV infection

anti-cancer;
anti-cancer
tumor suppressor genes
expressed
tumor suppressor genes
expressed
anti-cancer
suppresses organ
transplantation rejection

gout treatment
treat supraventricular
tachycardia; sleep regulation
(high: awake, low: asleep)
used for respiratory, UTI,
anthrax

anti-cancer

anti-cancer
anti-cancer

not used as an antibiotic

tuberculosis treatment

rifamycin family

causing intestinal problems

initially and later kidney and
liver failture; also contains
phalloidin binds actin and
inhibits MT depolymerization
in liver cells

from Death Cap mushroom

mRNA translocation by
GTPase blocked

antibiotic

antibiotic
antibiotic
meningitis treatment
lab tool
no clinical use

not used broadly; may inhibit

mito ribosomes
toxic to euk; prok and
mitochondria not effected
TOXIC to euk & prok; CTerminal puromycin causes
peptide to fall out of P-site
Decrease ATP Syn, Decrease
ETC & O2 consumption
Decrease ATP Syn, Decrease
ETC & O2 consumption
Decrease ATP Syn, Decrease
ETC & O2 consumption
Decrease ATP Syn, Decrease
ETC & O2 consumption
Decrease ATP Syn, Decrease
ETC & O2 consumption
Decrease ATP Syn, Decrease
ETC & O2 consumption
Decrease ATP Syn, Decrease
ETC & O2 consumption
Decrease ATP Syn, Decrease
ETC & O2 consumption
Decrease ATP Syn, Decrease
ETC & O2 consumption

Decrease ATP Syn, Increase

ETC & O2 consumption
Decrease ATP Syn, Increase
ETC & O2 consumption

Decrease ATP Syn, Increase

ETC & O2 consumption

dissipates proton gradient Decrease ATP Syn, Increase

ETC & O2 consumption

dissipates proton gradient Decrease ATP Syn, Increase

ETC & O2 consumption

Type/Name

Glycogen Storage Diseases

Enzyme Affected
Primary Organ

Type 0

Glycogen Synthase

Liver

Type 1 - Van
Gierkes's

G6Phosphatase deficiency

Liver

Type 2 - Pompe's
Disease

Lysosomal -1,4 & -1,6

glucosidase (acid matlase)
deficiency

skeletal & cardiac muscle

Type 3 - Cori's or
Forbe's (Limit
Dextrinosis)

-1,6 glucosidase
debranching enzyme
deficiency

liver, skeletal & cardiac

muscle

Type 4 Andersen's
Disease

Absence of branching
enzyme glucosyl 4:6
transferase

liver, muscle

Type 5 - McArdle's Absence of muscle

Syndrome
phosphorylase

skeletal muscle

Type 6 - Her's
Disease

liver

Type 8

deficiency of liver
phosphorylase
deficiency of liver
phophorylase kinase

liver, muscle

Manifestations
hypoglycemia, early death,
hyperketonia
liver cells & renal tubule cells
loaded w/glycogen.
Hypoglycemia, ketosis,
hyperlipidemia. Kidney failure
& thrombocyte dysfunction
Accumulation of glycogen in
lysosomes. Infantile form =
death by 2; Juvenile form =
myopathy; Adult form =
muscular dystrophyesque
Accumulation of a
characteristic branched
polysaccharide. Infant
hepatomegaly, myopathy
Accumulation of a
polysaccharide having few
branch points. Hepatomegaly,
cirrhosis. Death due to cardiac
or liver failure in 1st year of
life
Diminshed exercise tolerance.
Muscles have high glycogen
content, little or no lactate in
blood after exercise. Cramps,
pain, myoglobinuria
High glycogen in liver.
Hepatomegaly, mild
hypooglycemia,
hyperlipidemia, ketosis.
Improves w/age
like 6

Disease
Chagas
Leishmaniasis
Editing Glutamate Receptor in Brain
RNA Editing in Mammals
B-Thalasemmia
Limb Girdle Muscular Dystrophy
Systemic Lupus Erythematosis
Alzheimers
Mad Cow
Creutzfeld-Jacob
Scrapie
Cholera & E.Coli
Pertussis
Leigh Disease
Lactic Acidosis
Hypoglycemia
Essential Fructosuria
Hereditary Fructose Intolerance
Diabetes Mellitus
Classical Galactosemia
Non-Classical Galactosemia
Korsakoff/Wetberi berri
hereditary mitochondrial diseases
Lebers
Kearns-Sayre
MELAS
MERRF
ETC disorders
Prion disease
Ehlers-Danlos syndromes (EDS)

MO
RNA Editing via Guide RNA/Complex
RNA Editing via Guide RNA/Complex
Glutamine to Arginine in mRNA altering Ca2+ permeability via Adenosine Deaminase
Apo-B Gene; Cytidine Deaminase enters a stop codon (B-100-liver; B-48-Intestine)
Mutations in Globin gene causing additional splice sites w/in mRNA
Mutation in Calpain 3 gene generating new splice site at 16 = defective protein
Produce antibodies that react with U1 RNA component of splicosome
Diseased proteins stable; accumulate; damage brain
Brain develops holes from Prions which contain no nucleic acid; alter PrPc-->PrPsc via beta sh
Brain develops holes from Prions which contain no nucleic acid; alter PrPc-->PrPsc via beta sh
Brain develops holes from Prions which contain no nucleic acid; alter PrPc-->PrPsc via beta sh
ADP-ribosylate Galpha causing the inactivation of GTPase in Adynylate Cyclase
same as above just for Gi
Pyruvate DH deficiency-->Lactic Acidosis
Occursincreased NADH/NAD+, Thiamine deficiency -> decreased PDH; defect in gluconeo, d
due to UNRIPE ACKEE FRUIT-->inhibits beta-oxidation which doesn't produce acetylDeficiency in Fructokinase-->fructose excreted in urine; asymptomatic and abnormal dipstick
Deficiency in Aldolase Bstuck w/increased F-1-Ptrapping pi, inhibiting gluconeogenesi & g
high blood glucose-->sorbitol via aldosase reductase; can cause cataracts
high blood galactose-->galactitol via aldosase reductase; can cause cataracts; deficiency in G
deficiency in galactokinase
thiamine deficiencys-->ataxia, memory loss, cerebral hemorrhage

iseases
defect in NADH Dehydrogenase (COMPLEX I OF ETC)-->blindness
Mitochondrial myopathy, encephalopathy, lactic acidosis, stroke-like
Myoclonic epiplepsy, ragged red fibers

encepthalopathy due to abnormal change from alpha-helix to beta-sheet; secondary structure

mutation of the pro--chain gene (affects mainly type III collagen); also from collagen synth

Organism
Trypanosome
Leishmania

TSE; Transmissible Spongiform Enephalopathy

TSE; Transmissible Spongiform Enephalopathy
TSE; Transmissible Spongiform Enephalopathy
massive liquid secretion-->life threatening diarrhea

creased blood supply-->anaerobic in peripheral tissues

oa needed for pyruvate carboxylase needed for GLUCONEOGENESIS

cogenolysis-->hypoglycemia & jaundice

1-P URIDYL TRANSFERASE; stuck w/increased G-1-Ptrapping pi, inhibiting gluconeogenesis & glycogen

is enzyme deficiency

ogenesis & glycogenolysis-->hypoglycemia & jaundice

Diseases
Guillain Barre
Multiple Sclerosis
Parkinson's
Myasthenia Gravis
Hirschsprung
Barret's Esophagus
Pernicious Anemia
Peptic Ulcer
Celiac Disease
Acute Apendicitis
Tay-Sachs
Zellweger Syndrome
Rickets/Osteomalacia
Vitamin A
Osteoarthritis
Osteopetrosis
OI
Osteoporosis
Duchenne Muscular Dystrophy
Chromatolysis
wallerian degeneration

MO
Schwann's destroyed via large accumulation of lymphocytes, macrophages & plasma cells in Axon nerve fibers
Myelin detached from axon and destroyed; destruction of Oligodendrocytes
Loss of Dopamine secreting cells in Substantia Nigra & Basal Ganglia; Increase in Glial cells; Presence of Lewy B
Autoimmune attack on acetylcholine receptors; extra occular muscles usually first to go
Congenital MEGA COLON at RECTOSIGMOIDAL JNX; mutations in 1-4 genes
Metaplasia in cells of lower via acidic damage from stomach; Stratified Squamous--> Simple Columnar
Loss of Parietal Cells --> inadequate Intrinsic Factor --> Vitamin B12 not absorbed in Ileum
Destruction of mucosa, submucosa, muscularis via helicobacterium pylori --> scar tissue
Loss of Villus in small intestine; malabsorption
Early - Ulceration of Mucosa; Pus within lumen; vague abdominal pain
Late - Necrosis of muscularis; perforation-->Peritonitis; pus in peritoneal cavity-->septicemia, shock, death
lysosomal storage deficiencies-->accumulation of undigested substrate in lysosomes
deficiencies in peroxisomes that cause abnormalities in myelination of nerve cells
Vitamin D deficiency which is needed for ossification-->causes resorption
bone formation - deficiency; small stature - excess
joints wear out-->fluid build up
increased osteoblasts
type I collagen
critical loss of bone mass due to GH or Estrogen deficiency
Dystrophin - anchors fiber to lamina-->fragility and distructionreplaced by fibrous tissue
loss of Nissl from cell body - 1/2 days
anterograde on neuron.some retro but only few internodal segments..pns/days, cns/weeks

Other
Demyelinating; loss of muscle function - ascending motor paralysis
1. Myeling breakdown; 2. Proliferation of Astrocytes; 3. Reduced cellularity
Treat w/ L-Dopa
Arrest of neural crest cell migration to 1 segment of distal colon, ENS absent
Metaplastic cells are more prone to malignancy

can erode into peritoneal cavity --> peritonitis & if any blood vessels near erosion site, they'll become e

Disease
Patent Ductus Arteriosus
Coarctation of Aorta
Annular Pancreas
Omphacele
Umbilical Hernia
Gastroschisis
Meckel's Diverticulum
Erb's Palsy - C5/C6
Klumpke's Palsy - C8/T1
Acromioclavicular Separati
Nursemaid's Elbow
Pott's Disease
Ankylosing Spondylitis

MO
Failure of TGF-B induction after birth-->doesn't close--->Down Syndrome, Coarctation of Aorta
Extenions of Ductus Arteriosus muscles into Aorta causing pinching-->Turner Syndrome, Uppe
Bifid ventral pancreatic duct encircling duodenum causing obstruction

Damage to Upper Brachial Plexus-->Waiter's Tip - Arm:adducted/internally rotated; Forearm:p

Damage to Lower Brachial Plexus-->Claw/Ape Hand - Loss of intrinsic hand muscles (ulnar & m
Shoulder Elevates once corococlavicular ligaments tear as well
Subluxation of Radial head from ANNULAR ligament
"Tuberculous vertebral osteomyelitis" - pneumonia spread to spineinflammation
squaring of vertebra - bamboo spineo

Side Notes

Obstetrician/Motorcycle
Obstetrician/Cervical Rib
fall on tip of shoulder