stem cell research 101 · somatic cell nuclear transfersomatic cell nuclear transfer (scnt)...

Stem Cell Research 101

Stem Cell Research

Stem Cell Research 101: A promising type of bioscience research

The Stem Cell debate and the impact of The Stem Cell debate and the impact of the induced pluripotent stem cell procedureprocedure


Why Is Vocabulary Important?y y p

K t f th d b t Key terms frame the debate “Mature” versus “adult” stem cells “Early” versus “embryonic” stem cells Early versus embryonic stem cells “Therapeutic” versus “reproductive” cloning

DefinitionsMultipotent — Give rise to multiple but limited cell

typesPluripotent — Flexible, give rise to any cell type in

the body


y

What Is a Stem Cell? Unspecialized cells Give rise to more

than 250 pe i li ed ell inspecialized cells in

the body Serve as the Serve as the

body’s repair systemsystemRenew itselfReplenish other


pcells

Mature Stem Cells (MSC)

What Are the Sources of Mature Stem Cells?Mature Stem Cells?

Mature Body TissuesMature Body TissuesBrain

Neural cellseu a ce s

Heart

Cardiac muscle

Bone marrow


Blood cells

What Is a Treatment Example?p


Umbilical Cord & Placenta SC Research

l d Isolated immediately following birthg

Wharton’s Jelly showing promiseas a sourceas a source

More flexibility = some pluripotent p pcharacteristics

Research is limited but growing


but growing

Early Stem Cells (ESC)

What Are the Sources of Early Stem Cells?y


Where Are Early Stem Cells Found?y

5 days ofWho did it first? ydevelopment

Who did it first? In 1998,

U. WisconsinU. Wisconsin research team isolates stem cells from IVF-blastocysts


How Big Is a Blastocyst?g y


What Are the Characteristics?

Early stem a y stecells are pluripotentR i h Retain the special ability toability to develop into nearly

llany cell type


Somatic Cell Nuclear TransferSomatic Cell Nuclear Transfer (SCNT)

Sometimes referred to as “therapeutic cloning”

SCNT: Asexual Reproductionp

No sperm involved Transfers nucleus from a mature cell into a donor egg Requires electric or h i l ti l tchemical stimulus to

begin dividingF nctionall Functionally

different from regular fertilized egg


fertilized egg

How Would SCNT Treat Disease?


What Should You Know About SCNT?

Purpose:Purpose: Find cures and therapies for diseasesAwaken the natural capacity for self-repairAwaken the natural capacity for self repair

that resides in our genes Potential Results:Patients will receive own stem cells to

treat diseaseNo need for donor match

• Like transplantation, but without rejection


What Should You Know About SCNT?

No reputable scientist wants to clone No reputable scientist wants to clone human beings SCNT stem cells are alive in a Petri dish SCNT stem cells are alive in a Petri dish Unless implanted in uterus, you can not

t f tgenerate a fetus Evidence suggests that SCNT stem cells

may never develop into a human even when implanted


Most Recent Discovery

Induced pluripotent stem cell 2006/2007 - mouse and

human induced pluripotent stem cells were produced.

Patient

Introduce 3/4 genes found in stem cells into adult fibroblast

ll

Patient

cells

Ethical concerns eliminated, b t th tibut therapeutic concerns remain


Most Recent Discovery

Adult/Mature“Embryonic” Stem Cells

(Induced PluripotentAdult/Mature (skin) Cells

(Induced Pluripotent Stem – iPS)

Addition of Genes


Curing Mice of Sickle Cell Anemia

• Remove sickle cell DNA

• Replace with properMarrow cells

producing blood cellsSkin cells from mice

with sickle cell anemia

• Replace with proper genetic code

• Induce genetic change to bone

producing blood cells free of sickle cell

mutation

change to bone marrow cells

• Infuse each mouse with corrected

iPS cells

with corrected marrow cells made from its own skin cells


Public Policy and Ethics

How Is Stem Cell Research Funded and Regulated?

Both Kinds of SCR are Needed

Mature Stem Cells Early Stem CellsMature Stem Cells Early Stem Cells

First isolated in 1960s First isolated in 1998

~50 years of research 10 years of research

Federal Funding = Federal Funding = g>$2.462 Billion (FY04-FY07)

g$144 Million (FY04-FY07)

Results: 50+ human Results: Only in animalResults: 50+ human therapies

Results: Only in animal trials; No human trials


Who Funds Stem Cell Research?

NIH — All Stem Cell Research FY04 to FY07 = $2.462 Billion

NIH — Early Stem Cell Research NIH Early Stem Cell Research FY04 to FY07 = $144 Million

NIH Funding ($ in millions and rounded)

Research FY04 Actual

FY05 Actual

FY06 Actual

FY07 Actual

FY08 Estimate

FY09Estimate

Stem Cell Research

553 609 643 657 656 655

Human Embryonic Stem Cell Research

24 40 38 42 42 41


Source: NIH Budget Office (as of Feb. 2008)

Why Is Federal Funding Important?y g p

Most basic research supported by NIH fundsMost basic research supported by NIH funds Basic research requires millions of dollars

over many yearsover many years Difficult to find private funding for research

that is far from commercializationthat is far from commercialization It will be difficult for ESC research to

advance without federal fundingadvance without federal funding


Who Is Overseeing Research Ethics in the U.S.?g

National Academies of Science National Academies of Science Issued recommended guidelines in April 2005Scientific community developed guidelinesScientific community developed guidelines

• No government involvement

Purpose: enhance the integrity of privatelyPurpose: enhance the integrity of privately funded early stem cell research by encouraging responsible practicesg g p pUrged all institutions to establish oversight

committees; compliance is voluntary


; p y

What Are the Current Federal Restrictions?

Bush August 2001 gdecision

Limits federal funding to 78 t ll li li ibl78 stem cell lines eligible [global] 21 stem cell lines still

viable [global] Concerns about

contamination, ability to use in actual treatments, and long-term viability of stem cells


What Are the Current State Restrictions?

Current State Policies(as of January 2008)(as of January 2008)


Questions?Q


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