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Hemophilia

Alicia Song


Contents 01 02 03 04 05

What is Hemophilia? Inheritance Current Research Future Research What will the NIH money be used for?


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01 What is Title Hemophilia? Contents • When I find myself in times of trouble Mother Mary comes to me Speaking words of wisdom Let it be Hemophilia A: Not enough • And in my hour of darkness She is standing right in front of me Speaking words of wisdom Let It be Factor VIII • Let it be, Let it be, Let it be, Let it be Whisper words of wisdom Let it be

Hemophilia B: Not enough Factor IX Factor VIII, Factor IX: Clotting Factors

Factor VIII, Factor IX 1


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02 Inheritance Contents Title • When I find myself in times of trouble Mother to me Speakingfrom words sex of wisdom Let it be Recessive Inheritance from theMary X -comes chromosome chromosome • And in my hour of darkness She is standing right in front of me Speaking words of wisdom Let It be • Let it be, Let it be, Let it be, Let it be Whisper words of wisdom Let it be

Son: 0% Daughter: 100% to get carrier

Son: 50% Daughter: 50% to get carrier

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Son: 50% Daughter: 100% (usually die)


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03 CurrentTitle Research Contents • When I find myself in times of trouble Mother Mary comes to me Speaking words of wisdom Let it be

Transplant Stem Cell • And in my hour of darkness She is standing right in front of me Speaking words of wisdom Let It be

Because we are transplanting different person’s cell to the patient

• Let it be, Let it be, Let it be, Let it be Whisper words of wisdom Let it be

Problem: 1. Immune (Infection) 2. Need time and money

Stem cell 3 1


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04 Future Research Contents Title Claim: Future Research should be focused on • And in my hour of darkness She is standing right in front of me Speaking words of wisdom Let It be transplanting the normal stem cell by using • Let it be, Let it be, Let it be, Let it be Whisper words of wisdom Let it be CRISPR-Cas9

• When I find myself in times of trouble Mother Mary comes to me Speaking words of wisdom Let it be

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04 Future Research Contents Title • When I find myself in times of trouble Mother Mary comes to me Speaking words of wisdom Let it be How? • And in my hour of darkness She is standing right in front of me Speaking words of wisdom Let It be 1. Parent cells from patient. • Let it be, Let it be, Let it be, Let it be Whisper words of wisdom Let it be 2. Gene correction with CRISPR-Cas9. 3. Transplant.

Inversed gene cause Hemophilia

F VIII F IX Genes

Inversion – corrected gene

Do not cause Hemophilia

Use CRISPR-Cas9 to correct it

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04 Future Research Contents Title • When I find myself in times of trouble Mother Mary comes to me Speaking words of wisdom Let it be

Why CRISPR-Cas9? • And in my hour of darkness She is standing right in front of me Speaking words of wisdom Let It be • Let it be, Let it be, Let it be, Let it be Whisper words of wisdom Let it be

1. No immune problem. 2. Reduce time and money to treat.

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05 What will the NIH money be used for? Contents Title • When I find myself in times of trouble Mother Mary comes to me Speaking words of wisdom Let it be • And in my hour of darkness She is standing right in frontTeam of me Speaking words of wisdom Let It be Engineer • Let it be, Let it be, Let it be, Let it be Whisper words of wisdom Let it be

Building Materials, Facility for experiment

Resources

Raw materials 2 for researching CRISPR – Cas9

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Medical Equipment for Treating Patients


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Cited Images Contents Title

Cited Works

Azevedo, Margarida. “Hemophiliac Boy Who Had Successful Stem Cell Transplant Reported.” Hemophilia • When I find https://news myself in times of trouble Mother http://scie Mary comes News to meToday Speaking words , 1 Sept. 2016,of wisdom Let it be ela.com/read nceon.hani. /overview• And in my hour of darkness She is standing right in front ofhemophilianewstoday.com/2016/09/01/successfulme Speaking words of wisdom Let It be co.kr/?mid geneallogenic-stem-cell-transplant-boy-hemophilia-a/ =media&act editing/id/21 • Let it be, Let292 it be, Let it be, Let it be Whisper words of wisdom Let it be http://www.ih tc.org/medica lprofessionals /blooddisorders/ble edingdisorders/he mophilia-aand-b/ http://www.gr aphicsbuzz.co m/graphics/c oagulazionegraphics2775ce.html https://endo metriosisnew s.com/2016 /02/26

http://scie nceon.hani. co.kr/8429 6

https://v icare.vn/ baiviet/benh -mau-khodong-ditruyennhu-thenao/

Inacio, PhD Patricia. “CRISPR/Cas9 Gene-Editing Used in Hemophilia B Mice.” Hemophilia News Today, 5 Dec. 2016, hemophilianewstoday.com/2016/12/05/crisprcas9gene-editing-strategy-successfully-used-hemophilia-bmice/

http://www .neurologya dvisor.com/ neuropathic pain/diabet icperipheralneuropathyneuropathic -pain-scn9ageneticvariants/art icle/45203 4/

Kopperud, Daniel. “A Gene-Sequence Swap Using CRISPR to Cure Hemophilia.” Institute for Basic Science, 24 July 2015, www.ibs.re.kr/cop/bbs/BBSMSTR_000000000738/sel ectBoardArticle.do?nttId “Researching a Treatment for Hemophilia.” Wake Forest School of Medicine, 8 Aug. 2016, www.wakehealth.edu/Research/WFIRM/Projects/Rese arching-a-Treatment-for-Hemophilia.htm Cited 1

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Nih hemophilia  
Nih hemophilia  
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