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    Stem Cells andRegenerative

    Medicine

    Zach Scheiner,PhD

    Science Ofcer

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    What are stem cells? What arethe di erent types o stem cells

    Why should we study stemcells? What potential therapies

    co!ld they provide

    What are some o the challengesacing stem cell research

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    "ll Stem Cells

    Can#$% Sel &Rene'

    (% Di erentiate

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    Sel &Rene'al )symmetric celldivision*

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    S+in Progenitor Cell

    S+in Cell

    e!ral Progenitor Cell

    e!ron )-rain Cell*

    Stem CellEmbryonic

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    Stem Cell .ypes• /m0ryonic 1 pl!ripotent2 can

    ormalmost any cell type in the h!man

    0ody

    • .iss!e&Speci3c )"d!lt* 1m!ltipotent2 can orm only limitedtypes o cells )0lood, 0rain, liver,

    etc%*

    •4nd!ced Pl!ripotent

    1en ineered

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    Conception in a Dish4n 5itro 6ertili7ation

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    Day 1

    4n 5itro 6ertili7ation

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    Day 1

    4n 5itro Development

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    Day 2

    4n 5itro Development

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    Day 2

    4n 5itro Development

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    Day 3

    4n 5itro Development

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    Day 4

    4n 5itro Development

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    EmbryonicStemCells

    Day 5

    4n 5itro Development

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    This cell can form all of the

    cells in the human body

    This cell can formalmost every cell ty e

    in the human body

    !ully mature

    ll

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    8!man em0ryonic stem cells inc!lt!re

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    6l!orescent imaging oh!man em0ryonic stem cell

    colonies

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    What Diseases Do StemCells .reat 8ave the

    Potential to .reatC!rrently .reat

    & -lood Diseases )incl!dingimm!ne system disorders*& 9enetic meta0olicdisorders (verylimited/experimental)

    & .iss!e:organ replacement(very limited/experimental)

    Potential to .reat

    & 8eart Disease& e!rological Diseases)Par+inson;s, "l7heimer;s,8!ntington;s < others*& Stro+e

    & .ype $ Dia0etes& Mac!lar Degeneration )acommon ca!se o 0lindness*& Cancer& 845:"4DS& Spinal Cord 4n=!ry& M!ltiple Sclerosis& ;

    Potential to .reat

    & 8eart Disease& e!rological Diseases)Par+inson;s , "l7heimer;s,8!ntington;s < others*& Stro+e

    & .ype $ Dia0etes& Mac!lar Degeneration )acommon ca!se o 0lindness*& Cancer& 845:"4DS& Spinal Cord 4n=!ry& M!ltiple Sclerosis& ;

    "andmore#

    C!rr ently .reat

    & -lood Diseases )incl!dingimm!ne system disorders*& 9enetic meta0olicdisorders (verylimited/experimental)

    & .iss!e:organ replacement(very limited/experimental)

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    -one Marro' )8ematopoietic Stem Cell* .ransplant

    /?ample o a tiss!e&speci3c stem cell therapy

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    .rachea transplantation/?ample o tiss!e&speci3c stem cell&0ased tiss!e

    replacement

    $lic% on Picture to Play &ideo

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    .iss!e&speci3c )ad!lt* stem

    cells are po'er !l andpromising@Why do researchers study

    embryonic stem cells?• .iss!e&speci3c stem cells are limitedin their di erentiation potential )0lood

    0lood*•Stem cells rom some tiss!es areinaccessi0le•Some tiss!e&speci3c stem cells don;t

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    /m0ryonic Stem&Cell

    Derived 8eart M!scleCells

    $lic% on Pictures to Play &ideos

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    /m0ryonic Stem

    Cells

    5ideo2 Dia0etes " C4RM Disease .eam

    5ideo2"ge&Related Mac!lar Degene

    ration )"MD*

    http://diabetes_final_1280x720.mov/http://amd_cirm.mov/http://amd_cirm.mov/http://amd_cirm.mov/http://amd_cirm.mov/http://diabetes_final_1280x720.mov/

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    5ideo2 C4RM Disease .eam or .ype $Dia0etes

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    5ideo2 C4RM Disease .eam or "ge&Related Mac!lar Degeneration )"MD* 1

    /ye Disease

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    What are some o thechallenges acing

    em0ryonic stem cellresearch

    • Di erentiation o stem cellsinto

    mat!re, !nctional cells

    • Potential or t!morormation

    4 d! d Pl! i S )iPS*

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    4nd!ced Pl!ripotent Stem )iPS*Cells

    9enetically engineering ne' stem cells

    Skin cells iPS cells

    Pros2 o em0ryosreA!ired o imm!nere=ection Disease in a dish

    Cons2 May not 0e B to/SCs

    9eneticallyen ineered

    5ir!sengineeredto e?press o!r+ey

    pl!ripotencygenes

    5id 2 P +i di &i &

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    5ideo2 Par+inson;s disease&in&a&dish

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    "c+no'ledgements& .odd D!0nico & "my "dams& >a!rel -archas

    www.cirm.ca.gov

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    /?tra Slides

    ! l ll d !

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    e!ral stem cells or dr!gdelivery

    6oc!sed delivery o chemotherapy or cancer

    NSCsinjected

    (no tumor)

    NSCsinjected(tumor)

    Day 0 Day 7 Day 14

    Shah et al. Dev Neurosci 2004

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    Stem cells or dr!g delivery6oc!sed delivery o chemotherapy or cancer

    "nother C4RM Disease .eam

    enetically En!ineeredNeural Stem Cells

    "ice are !i#en a non$to%ic dru!& 'hich theneural stem cells cancon#ert to an acti#e dru!to shrin tumors

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    Milestones in /m0ryonicDevelopment

    • Days E&$F2 /m0ryo implants in the !ter!s• Day $F2 .hree distinct layers 0egin to

    orm )no more em0ryonic stem cells*

    • Days $F&($2 -eginning o !t!re nervo!ssystem

    • Days ($&(F2 -eginning o !t!re head,

    nec+, mo!th, and nose• Wee+s G&H2 -eginning o organ ormation• Wee+ H2 /m0ryo is called a et!s