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Cloning & stem cell strategies in transplantation
Robert Lanza, MDVP Medical & Scientific DevelopmentAdvanced Cell Technology
Adjunct ProfessorInstitute for Regenerative MedicineWake Forest University School of Medicine
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Alzheimer ’s
Dwarfism
Parkinson ’s
Strokes
Epilepsy
Hemophilia
Kidney failure
Chronic pain
CancerInfertility
Burns
AIDS
Muscular dystrophy
ALS
Affective disorders
Macular degeneration
Hypoparathyroidism
Heart disease
Liver failure
Enzymatic defects
Diabetes
Osteoarthritis
Multiple sclerosis
Huntington ’s
Hypocholesterolemia
Rheumatoidarthritis
Atherosclerosis
Ulcers
Spinal cord injuries
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2001Year
Num
ber
of P
atie
nts (
in th
ousa
nds)
Waiting List
Organs Transplanted
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2001
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2003 [First stem-cell stage SCNT embryos]
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Lanza et al Science 2000Lanza et al Science 2000
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0.0
20.0
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80.0
0.0 1.0 2.0 3.0 4.0 5.0 6.0
A
.
Telomere fluorescence (in kMESF)
0.0 10.0 20.0 30.0 40.0 50.0 60.0 70.0 80.0
Dupl. 1Dupl. 2
Exp. 1 Exp. 2
Young cattle5-10 mos old
Old cattle> 10 yrs old
Newborn calves< 2 wks old
Cloned cattle5-10 mos old
10 20 30 40 50 60 70 10 20 30 40 50 60 700 80
Cy= - 3.62 x + 61.8
R2 = 0.54
B
Years
kMES
F
23.1
9.4
1 2 3 4 5 6 7 8 9 10
Mean TRF 18.3 15.2 20.1 18.3 15.2 20.1 14.4 16.4 12.1 16.1
kb
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0
20
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100P
OP
ULA
TIO
N D
OU
BLI
NG
S
NORMAL FETUS CLONED FETUS
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•Parkinson’s ($6 billion/yr)
•Stroke ($45 billion/yr)
•Spinal cord injury ($10 billion/yr)
•Epilepsy ($3 billion/yr)
•Alzheimer’s ($100 billion/yr)
•Multiple sclerosis ($10 billion/yr)
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Production of new hES lines that will be safe forclinical applications
• Feeder-free system• Serum-free system• Elimination of all
animal products,including sialic acids
• Generated using goodmanufacturingpractice (GMP)
A B
C D
E
G H
I
L
F
A B
C D
E
G H
I
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F
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Generation of ES cells using parthenogenesis
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Retinal Degenerative Diseases
• Age-related macular degeneration (ARMD) alone affects over 30 million people worldwide• Leading cause of blindness in patients over 60 yrs old in the US• Degeneration of retinal pigment epithelium (RPE) plays a critica l
role in the pathogenesis of AMRD as well as retinitis pigmentosaand other retinal degenerative diseases
• Transplanted RPE cells have been shown to rescue host photoreceptors and attenuate loss of visual function in animals
• Stem cell -derived RPE could be a potentially important source of tissue for subretinal transplantation in patients
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WBC colony from cloned stem cells
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•Cardiovascular disease costs the US $329 billion annually
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End-stage renal disease will cost US $1 trillion during the comingdecade
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