Use of Gene Editing Technologies in Rodents (Carlisle Landel)

8/20/2019 Use of Gene Editing Technologies in Rodents (Carlisle Landel)

1/19

Vsf ol

Nfef

Fmctcen

]fajeodoncfs

ce Pomfets

Agrdcsdf R. Dgemfd, Rj.M.


2/19

]jf Iousf

gs

G

Iomfd

• Igiigd

• Qigdd, fgsy

to

igcetgce,

lfauem

• ^fdd‒uemfrstoom nfeftcas

‑ Qcicdgrcty to juiges 532%

‑ Gvgcdg`cdcty ol ce`rfm strgces

‑ Jcnjdy gifeg`df to nfeoif iomclcagtcoe• ]jousgems ol strgces ol nfeftcagddy iomclcfm iousf

strgces fxcst

• @gehs ol FQ afdds wctj iutgtcoes ce gdiost gdd iousf nfefs fxcst


3/19

Icdfstoefs ce Iousf Nfeftcas

7321 Ifemfd–s dgws vfrclcfm ce igiigds uscen iousf aogt aodors

7302–s Nfeftca `gscs lor ageafr; tuior susafptc`df iousf strgces

7312–s Fxtrgajroiosoigd trgesicsscoe ol tuiors (vcrus, II]\)

73=2–s Jcstoaoipgtc`cdcty getcnfes igppfm (: dcehgnf nroups)

Fllfat ol utfrcef fevcroeifet oe `crtj mflfats fstg`dcsjfm

Iousf iutgets usfm to iomfd juige aoenfectgd mflfats

7362–s @codoncagd fllfats ol rgmcgtcoe gem ajficagd iutgnfes

73:2 ]rgesnfeca iousf tfajeodony; proeuadfgr cekfatcoe

73:= Cetfrspfacfs joiodoncfs ce dcehgnfs fvcmfet

73:< Fi`ryoeca stfi afdd tfajeodony; `dgstoayst cekfatcoe

7332–s FEV iutgnfefscs prokfats

0222 572,222 nfefs, 506,222 igrhfrs ce iousf0221 Iousf nfeoif mrglt sfqufeaf gvgcdg`df

022> Heoah-Out Iousf Rrokfat (HOIR)

0223 ]grnftfm Euadfgsfs aoif oedcef aoiifracgddy


4/19

]jf Pgt

gs

g Iomfd

• Dgrnfr

‑ Iorf fxpfescvf

to

igcetgce,

sdcnjtdy

doenfr

nfefrgtcoe tcif

‑ Iorf igtfrcgd pfr gecigd

• Rrflfrrfm lor toxcaodoncagd gem `fjgvcorgd stumcfs

• Qoif nfeftca

pjfeotypfs

iorf

adosfdy

icica

tjf

juige aoemctcoe aoipgrfm to icaf

• Gvgcdg`cdcty ol nfeoif iomclcagtcoe wgs

trgmctcoegddy iuaj

iorf

dcictfm

tjge

wctj

icaf

‑ Cectcgd trgesnfefscs gttfipts wfrf cefllcacfet

‑ Eo FQ afdds uetcd 0223


5/19

(O`tgce/promuaf FQ Afdds)

FQ Afdd‒Ifmcgtfm ]grnftfm

Iutgnfefscs(tjf `gm odm mgys)

Igecpudgtf FQ

afdds

ce

vctro

Csodgtf tgrnftfm adoefs

]cifdcef; =‒: wffhsCesfrt tgrnftfm adoefs ceto `dgstoaysts

]rgeslfr cekfatfm `dgsts to lostfr iotjfrs

Qarffe lor

ajcifrca

ollsprcen

agrrycen tgrnftfm iutgtcoe

]cifdcef; =‒: wffhs

Igtf

to

promuaf

jftfrozynous

nfridcef

louemfrs]cifdcef; : wffhs

Igtf to promuaf joiozynous iutget gecigds

]cifdcef; : wffhs

]otgd ]cifdcef ]o Joiozynous Iutgets; : ioetjs iceciui


6/19

]jf ]grnftfm

Euadfgsf

Lgicdy

• [LEs ‑ Lcrst gppdcfm to rgts ce 0223 gem to icaf ce 0272

‑ Mcsgmvgetgnfs;

fxpfescvf, eot

so

fgsy

to

aoestruat,

dcictfm tgrnft gvgcdg`cdcty, oefrous CR rfgaj‒tjrounj

• ]GDFEs (Z]Es) ‑ Pomfet gppdcagtcoes `fnge ce 0277

‑ Fgscfr to

ighf

tjge

[LEs,

age

tjforftcagddy

tgrnft

gey

sfqufeaf

• APCQRP/AGQ3

‑ Pomfet

gppdcagtcoes

`fnge

ce

0271 ‑ Fxtrfifdy fgsy to fenceffr, gifeg`df to iudtcpdfx tgrnftcen

‑ Iorf proef to oll ‒tgrnft iutgnfefscs, soifwjgt tgrnft‒rfstrcatfm


7/19

Mou`df‒Qtrgem

@rfgh

Pfpgcr

>

Eoe‒Joiodonous

Fem Kocecen

Joiodonous

Pfaoi`cegtcoe Pfpgcr

Frror‒Rroef

Cetromuafs sigdd mfdftcoes/cesfrtcoes

Frror‒Lrff


8/19

MEF

MEG `rfgh

aomcen sfqufeaf

aomcen sf qufeaf

iutgnfeca

rfpgcr `y

EJFK

aomcenqufeaf

MEF

MEG `rfgh

joiodonous

rfaoi`cegtcoe

pdgsicm MEG

agrno

agrno

MEF

MEG `rfgh

aomcen sffhwfeaf

aomcen sf fhwfeaf

aomcen sfqufeaf

joiodonous

rfaoi`cegtcoe

aomcen sfqufeaf

pdgsicm MEG

]grnftfm Euadfgsf

Iutgnfefscs


9/19

]grnftfm Euadfgsf‒Ifmcgtfm

Iutgnfefscs Rroafss

]rfgt 7 afdd fi`ryos wctj rfgnfet

]rgeslfr trfgtfm fi`ryos to lostfr iotjfrs

Qarffe lor

iutget

ollsprcen

]cifdcef;


10/19

Iousf Fi`ryo

Icarocekfatcoe


11/19

Mf`ut ol

APCQRP/AGQ3


12/19

Pfsudts ol

^gen

ft

gd.

• APCQRP tgrnftcen cs fxtrfifdy fllcacfet

‑ Gs jcnj

gs

722%

tgrnftfm

pups

gem

36%

joiozynous tgrnftcen lor soif gddfdfs/aoemctcoes

‑ :2% ol pups lroi mou`df‒nfef tgrnftcen wfrf

mou`df joiozynotfs


13/19

Cipdcagtcoes ol

^gen

ft

gd.

• Nfef tgrnftcen ce romfets cs eow fxtrfifdy

fgsy ‑ Cs FQ afdd‒ifmcgtfm tgrnftfm iutgnfefscs

o`sodftf?

• Qciudtgefous tgrnftcen ol iudtcpdf nfefs proicsfs trfifemous sgvcens ol tcif gem

gecigds


14/19

]rgmctcoegd Rromuatcoe ol Dcefs

Agrrycen ]wo

Iutgtcoes

Iut 7

wt

Iut 0

wt

Iut 7

wt

Iut 0

wt8

Iut 7

wt

Iut 0

wt8

Iut 7

wt

Iut 0

wt8

Iut 7

Iut 7

Iut 0

Iut 08

06% ol gecigds

:

wffh

proafss


15/19

]rgmctcoegd Rromuatcoe ol Dcefs

Agrrycen ]jrff

Iutgtcoes

Iut 1

wt

Iut 7

wt

Iut 0

wt8

70.6%

ol

gecigds

:

wffh

proafss

Iut 7

wt

Iut 0

wt8

Iut 1

wt8

Iut 7

wt

Iut 0

wt8

Iut 1

wt8

Iut 7

Iut 7

Iut 0

Iut 08 Iut 1

Iut 18

7.


16/19

]grnftfm Euadfgsf Iutgnfefscs Jgs

@ffe Vsfm

]o

Nfefrgtf;

• —Heoahout’ iutgtcoes, c.f., cegatcvgtf spfaclca

nfefs• —Heoahce’ iutgtcoes, c.f., cesfrt nfeftca igtfrcgd

gt g mflcefm nfeoica sctf

‑ Qcendf `gsf

ajgenfs

gt

spfaclca

sctfs

‑ Qigdd cesfrtcoes ol spfaclca sfqufeaf, f.n. rfaoi`cegsf tgrnfts dchf doxR, fta.

‑ ]rgesnfef cesfrtcoes gt —sglf sctfs’ or uemfr tjf aoetrod

ol

femonfeous

proiotfrs

• Dgrnfr sagdf mfdftcoes or cesfrtcoes


17/19

Pfigcecen Cssufs

• Oll ‒tgrnft iutgnfefscs

‑ Qystfi iomclcagtcoe

lor

cearfgsfm

lcmfdcty

‑ Ce tjf gecigd iomfd wordm, eot suaj ge cssuf; —^f–dd

kust `rffm tjfi out.’

• Aoevfrtcen —grt’

to

g routcef

iftjomodony

‑ ^jgt mflcefs g noom tgrnft? Eot gdd doac grf fqugd.

‑ Jow mo wf `cgs tjf systfi cemfds vs cesfrtcoes?

‑ ^jgt grf

tjf

`fst

rfgnfets/aoemctcoes/prgatcafs

lor

suaafss?


18/19

]grnftfm Euadfgsfs Jgvf

Pfvodutcoeczfm Iousf

Nfeoif

Fenceffrcen• Eo doenfr dcictfm `y FQ afdd gvgcdg`cdcty

‑ Gey strgce age (potfetcgddy) `f tgrnftfm

‑ Gey (romfet) spfacfs age `f tgrnftfm

• ]cifdcefs to

iomfd

nfefrgtcoe

jgvf

`ffe

scneclcagetdy sjortfefm

• Gecigd usf lor iomfd promuatcoe jgs

mfarfgsfm


19/19

Wufstcoes?

Agrdcsdf R.

Dgemfd,

Rj.M adgemfdBiga.aoi

Use of Gene Editing Technologies in Rodents (Carlisle Landel)

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