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Therapeutic Possibilities ofT

herapeutic Possibilities of

Stem Cell ResearchStem Cell Research

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Cystic Fibrosis: Stem Cell-C

ystic Fibrosis: Stem Cell-

Gene Therapy Approach:Gene Ther

apy Approach:

n Human bone marrown Human bone marrowderived stem cells canderived stem cells candifferentiate into airwaydifferentiate into airway

epithelial cellsepithelial cells n Encoding these cells withn Encoding these cells with

the gene that is defectivethe gene that is defectivein CF restores cellularin CF restores cellularfunctionfunction

n Keep airways clear ofn Keep airways clear ofmucus and air-bornemucus and air-borneirritantsirritants

n Hope to perform clinicaln Hope to perform clinicaltrial in next 2-3 yearstrial in next 2-3 years

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Biological Pacemaker:Bio

logical Pacemaker:

n Human Embryonicn Human EmbryonicStem Cells geneticallyStem Cells geneticallyengineered and coaxedengineered and coaxedto become heart cellsto become heart cells

n Clusters of cells beatn Clusters of cells beaton their own triggeredon their own triggeredthe unified beating ofthe unified beating ofrat heart muscle cellsrat heart muscle cells

n Triggered regularn Triggered regularbeating when implantedbeating when implanted

in guinea pigsin guinea pigs n Cells responded ton Cells responded to

drugs used to slow ordrugs used to slow orspeed up heart ratespeed up heart rate

n Use geneticn Use geneticengineering toengineering to

customize the pacingcustomize the pacingrate of the cellsrate of the cells

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Retinal Degeneration:Retinal De

generation:

n Mice predisposed forn Mice predisposed forRetinitis Pigmentosa: aRetinitis Pigmentosa: adegenerative disease thatdegenerative disease thatdestroys retinasdestroys retinas

n Injected bonen Injected bonemarrowderived stem cells intomarrowderived stem cells intothe back of mouse eyesthe back of mouse eyesduring developmentduring development

n Dramatically curtailedn Dramatically curtailedretinal degenerationretinal degeneration

n Completely normaln Completely normalvasculature, improved retinalvasculature, improved retinal

tissue and light responsetissue and light response n Disorders of the retina thatn Disorders of the retina that

have vascular and neuronalhave vascular and neuronaldegeneration: geneticdegeneration: geneticdisorders known collectivelydisorders known collectivelyas retinitis pigmentosaas retinitis pigmentosa

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Blueprintcells

Human Embryonic Stem Cells

Pipette

Stem Cells

To remove the stem cells, the Blastocyst is openedand the stem cells removed with a pipette

Blastocyst -from In Vitro Fertilization Clinic

Stem Cells Blueprint cells

A Blastocyst is a hollow ball of cells with a

small clump of stem cells inside

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Pipette

Pipette

Stem Cells

Petri Dish

Human Embryonic Stem Cells

To remove the stem cells, the Blastocyst is

broken open and the stem cells removed with

a pipette(an ultra thin glass tube)

The stem cells are

placed in adish and are fed and

cared for(each blastocyst =

1 stem cell line)

Blastocyst -from In Vitro Fertilization Clinic

Stem Cells Blueprint cells

A Blastocyst is a hollow ball of cells with a

small clump of stem cells inside

Stem Cells

Blueprint

cells

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Neuron

Muscle

cell

PancreaticIslet

Petri DishStem Cells

Different chemicals / molecules are added to the stem

cells to make them become specific types of cells.

Growth factors Chemical cues

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The vision for UCIs Stem CellCenter:Stem cell therapies for neurologicaldisorders

Brain and spinal cordinjury.

Stroke.

Neurodegenerativediseases Parkinsons Disease

Huntingtons Disease

Alzheimers Disease

Multiple Sclerosis

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Goal #2: To discover how to make stem cells

integrate into neural circuits.

Nerve cell (neuron) Oligodendrocyte

More research is needed to find the

ways to actually use stem cells for

therapeutic applications.

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1. They can generate large

quantities of tissue rapidly

2. They can become any cell in the bodyEmbryonic stem

cells

Brain Heart

Cartilage

Bone marrowFat

Embryonic Stem Cells

Human Embryonic Stem Cells