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LJGJ\NB

Muiu`s Lmjarnjt*, Jcc` Ofut`b*, Kjrtnc Gnlmijcs*, @bnzja`tm Wfurcn`r-Bjss`rv`,

Kjrn` I`rkjnc` Afuss`r

Wr`jtk`ct

Jt pr`s`ct, tm`r` ns cf tr`jtk`ct fh prfv`c `hhnljly hfr LJGJ\NB,`ntm`r hfr tm` gns`js` fr hfr tm` kjnc sykptfks. Wr`jtk`ct ns tmus`ctnr`by prjikjtnl.

Bjcl`t C`urf b155>4 =?87

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LJGJ\NB

Uft`ctnjb tm`rjp`utnl jv`cu`s6

Jcc` OFPW@B

Kjrd C@B\FC

7/22/2019 Anne Joutel and Mark Nelson - Potential therapeutic avenues ?

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Gns`js`-kfgnhynci tr`jtk`cts ajs`gfc ucg`rstjcgnci tm` pjtmfi`cnl s`qu`cl`

\trfd` lficntnv` $ kftfr gnsjanbnty

Ymnt` kjtt`r lmjci`s

Knirjnc` wntm jurj

Cftlm< @LG/ IFK g`pfsnts

~15 ~

7/22/2019 Anne Joutel and Mark Nelson - Potential therapeutic avenues ?

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Cftlm

7/22/2019 Anne Joutel and Mark Nelson - Potential therapeutic avenues ?

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Jctns`cs` fbnifculb`ftng`s (J\Fs)tf r`gulnci tm` lfcl`ctrjtnfc fh prft`ncs

Hrfk \futmw`bb `t jb, Wr`cgs nc Kfb`lubjr K`gnlnc` 15:1

\mfrt syctm`tnl lm`knljbby kfgnhn`g culb`nl jlngs tmjt ancg QCJ tjri`t

I`c` snb`clnci (g`irjgjtnfc) fr kfgubjtnfc fh QCJ k`tjafbnsk (spbnlnci)

Cfcs`b`ltnv` (YW &kutjct jbb`b`s) fr kutjct jbb`b`-sp`lnhnl snb`clnci

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Hjvfrjab` lmjrjlt`rnstnls Bfci tnssu` mjbh-bnh` fh lm`knljbby kfgnhn`g (ajldafc` strultur` fr suijr rnci ) J\Fs

(~1= gjys)

@hhnln`ct LC\ g`bnv`ry ay nctrjtm`ljb nchusnfc nctf tm` L\H

@hhnln`ct uptjd` nc c`urfcs jcg ibnj (\KL66)

Culb`jr `ctry

Cjd`g J\Fs 3 ir`jt`r tfb`rjcl` jcg pr`lns` gfsnci

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A`c`hnlnjb `hh`lt kjy p`rsnst hfr bfci`r tmjc tjri`t suppr`ssnfc 3 trjcsn`ct

tm`rjpy

J pmjs` : hnrst-nc-kjc w`bb-tfb`rjt`g stugy (\FG:-JB\) (pmjs` 1 nc WWQ-jkybfngfsns)

Jctns`cs` fbnifculb`ftng`s (J\Fs) tm`rjp`utnlshfr ncm`rnt`g arjnc gns`js`s

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Jctns`cs` fbnifculb`ftng`s tm`rjp`utnls hfr LJGJ\NB6

Q`gul` `xpr`ssnfc fh jbb Cftlm

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Jkybfng ljsljg` mypftm`sns (tfxnlnty fh sfbuab`, fbnifk`rnl jcg hnarnbbjr Jsp`ln`s)

Ujssnv` (Jctn-J jctnafgn`s jgkncnstrjtnfc) / jltnv` (J nkkucnzjtnfc tfncgul` jctn-J nkkuc` r`spfcs`)

Jtt`cujt`s J g`pfsntnfc jcg hjlnbntjt`s lb`jrjcl` fh J jiir`ijt`s nckfus` kfg`bs jcg pjtn`cts wntm Jbzm`nk`rs gns`js`

Wftjb LC\ `xpfsur` fh jctn-J jctnafgn`s g`p`cgs fc hrjltnfcjb LC\p`c`trjcl` (5.50-5.1%) + LC\ mjbh bnh` (5.0-7 mfurs)+ lylbnci a`tw``c LC\jcg pbjskj+ pbjskj mjbh bnh` (:-< w``ds)+ anfjvjnbjanbnty

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Wm` jctn-J nkkucftm`rjpy nc Jbzm`nk`rs gns`js`?:7 y`jrs fh `xp`rn`cl`

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Wm` lmjbb`ci` fh jctn-J nkkucftm`rjpy jgv`rs` `hh`lts

Hrfk Bnu, .-M. `t jb., Cjt. Q`v. C`urfb. 15:

Js`ptnl

k`cncif`cl`pmjbntns

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7/22/2019 Anne Joutel and Mark Nelson - Potential therapeutic avenues ?

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Wm` hjnbur`s fh pmjs` NNN jctnafgy trnjbsnc Jbzm`nk`rs gns`js` - Hutur` gnr`ltnfcs

Q`jsfcs hfr hjnbur` Inv`c tff bjt`

Ujrtnlnpjcts wntmfut jacfrkjb jkybfng aurg`c ay U@W sljccnci (~

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@xtrjl`bbubjr Cftlm