Lysosomes are membrane-enclosed organelles that

contain an array of enzymes capable of breaking

down all types of biological polymersproteins,
nucleic acids, carbohydrates, and lipids. Lysosomes
function as the digestive system of the cell, serving
both to degrade material taken up from outside the
cell and to digest obsolete components of the cell
itself.
When viewed through a microscope lysosomes are
visualized as dense spherical vacuoles, but they can
display considerable variation in size and shape as a
result of differences in the materials that have been
taken up for digestion.
(change slide)
Primary lysosomes have not started a digestive event
Secondary lysosomes have fused with the membrane
of a phagosome
Tertiary lysosomes are left over particles from
incomplete digestive events. These can be seen in
various organs and are known as aging pigment
(change slide)
Lysosomes contain about 50 different enzymes that
can hydrolyze proteins, DNA, RNA,
polysaccharides, and lipids.
All of the lysosomal enzymes are acid
hydrolases
Acid Hydrolases is an enzyme that speeds
up the process of hydrolysis.
Acid hydrolases are active at an acidic pH
of about 5 that is maintained within
lysosomes but not at the neutral pH
(about 7.2) characteristic of the rest of
the cytoplasm. This is important when
discussing the effects of Chloroquine
(change slide)
This slide shows the two ways which
lysosomes function
Autophage- lysosome digestion of self
Heterophage- Lysosome digestion of
foreign substance
(change slide)
Chloroquine, as an agent used in the treatment and
prevention of malaria.
Cholloquine raises the pH of the lysosomal content,
thereby inactivating many lysosomal enzymes. The
action of chloroquine on lysosomes accounts for its
antimalarial activity. The drug concentrates in the
acidic food vacuole of the malaria parasite
(Plasmodium falciparum) and interferes with its
digestive processes, eventually killing the parasite.
(change slide)
So why else are these lysosomes important? In
addition to their function for the body, mutations in
the genes that encode these enzymes are responsible
for more than 30 different human genetic diseases.
For example, Gauchers disease (the most common
of these disorders) results from a mutation in the
gene that encodes a lysosomal enzyme required for
the breakdown of glycolipids.
The lack of the glucocerebrosidase enzyme causes
harmful substances to build up in the liver, spleen,
bones, and bone marrow. The substances prevent
cells and organs from working properly.
Treatment
Enzyme replacement therapy is available. A bone
marrow transplant may be needed in severe cases.

Hurler syndrome is a rare, inherited disease of

metabolism in which a person cannot break down
long chains of sugar molecules called
glycosaminoglycans
Persons with Hurler syndrome do not make a
substance called lysosomal alpha-L-iduronidase.
This enzyme, helps break down long chains of sugar
molecules called glycosaminoglycans Without the
enzyme, glycosaminoglycans build up and damage
organs, including the heart. Symptoms can range
from mild to severe.
Hurler syndrome is inherited.
Treatment
Enzyme replacement therapy for Hurler syndrome
adds a working form of the missing enzyme to the
body. The medication, called laronidase
(Aldurazyme), is given through a vein
(intravenously).
Bone marrow transplant has been used in several
patients with this condition. The treatment has had
mixed results.
Other treatments depend on the organs that are
affected.

Sanfilippo syndrome
Sanfilippo syndrome is an inhertited disorder that
makes the body unable to properly break down long
chains of sugar molecules called
glycosaminoglycans. (just like Hurlers)
Sanfilippo syndrome occurs when the substances
(enzymes) needed to break down the heparan sulfate
sugar chain are missing or are defective.
There are four main types of Sanfilippo syndrome,
also called MPS III. Which type a person has
depends on which enzyme is affected.

Sanfilippo type A is the most severe

form.
There is no specific treatment available for
Sanfilippo syndrome.
Outlook (Prognosis)
The syndrome causes significant neurological
symptoms, including severe intellectual disability.
IQs may be below 50. Most persons with Sanfilippo
syndrome live into their teenage years. Some
patients live longer, while others with severe forms
die at an earlier age. Symptoms appear most severe
in persons with type A Sanfilippo syndrome.

Tay-Sachs disease
Tay-Sachs disease is a life-threatening disease of the
nervous system passed down through families.
Causes, incidence, and risk factors
Tay-Sachs disease occurs when a protein that helps
break down a chemical found in nerve tissue called
gangliosides. Without this protein, gangliosidesbuild
up in cells, especially nerve cells in the brain.
Tay-Sachs disease is caused by a defective gene on
chromosome 15.
Tay-Sachs has been classified into infantile, juvenile,
and adult forms, depending on the symptoms and
when they first appear. Most people with Tay-Sachs
have the infantile form. In this form, the nerve
damage usually begins while the baby is still in the
womb. Symptoms usually appear when the child is 3
to 6 months old. The disease tends to get worse very
quickly, and the child usually dies by age 4 or 5.
Late-onset Tay-Sachs disease, which affects adults,
is very rare.
Eye exam (reveals a cherry-red spot in the macula)
Treatment
There is no treatment for Tay-Sachs disease itself,
only ways to make the patient more comfortable.