Katherine A. High

Katherine A. High
Katherine A. High
Born	High Point, North Carolina
Nationality	American
Alma mater	Harvard College, University of North Carolina School of Medicine
Known for	Gene Therapy
Children	three, including Sarah Steele
	Scientific career
Fields	Hematology
Institutions	University of Pennsylvania, Spark Therapeutics
Academic advisors	Edward J. Benz Jr.

Katherine A. High is an American doctor-scientist who is an emeritus professor at the Perelman School of Medicine at the University of Pennsylvania. She was the co-founder, president, and chief scientific officer of Spark Therapeutics and currently serves as President of Therapeutics at AskBio.^[1] She has worked in the area of gene therapy, performing both basic research and clinical investigations. She has been recognized for her distinguished contributions to the field, having designed, sponsored, and conducted the first clinical trial of an adeno-associated viral vector (AAV) gene therapy injected into the skeletal muscle (1999),^[2] the first trial of AAV gene therapy introduced into the liver (2001),^[3] and the first trial in the US of an AAV gene therapy injected into the subretinal space (2007).^[4]

^ Yarbrough, Cathy (3 September 2018). "Gene Therapy Pioneer's 30 Years Of Problem-Solving". Life Science Leader.
^ Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA (2000). "Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector". Nature Genetics. 24 (3): 257–261. doi:10.1038/73464. PMID 10700178. S2CID 12650196.
^ Manno CS, Arruda VR, Pierce GF, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt, P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA (2006). "Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response". Nature Medicine. 12 (3): 342–347. doi:10.1038/nm1358. PMID 16474400. S2CID 14664182.
^ Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J (April 28, 2008). "Safety and efficacy of gene transfer for Leber's Congenital Amaurosis". New England Journal of Medicine. 358 (21): 2240–2248. doi:10.1056/NEJMoa0802315. PMC 2829748. PMID 18441370.

[:0-1] Yarbrough, Cathy (3 September 2018). "Gene Therapy Pioneer's 30 Years Of Problem-Solving". Life Science Leader.

[2] Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA (2000). "Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector". Nature Genetics. 24 (3): 257–261. doi:10.1038/73464. PMID 10700178. S2CID 12650196.

[3] Manno CS, Arruda VR, Pierce GF, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt, P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA (2006). "Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response". Nature Medicine. 12 (3): 342–347. doi:10.1038/nm1358. PMID 16474400. S2CID 14664182.

[4] Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J (April 28, 2008). "Safety and efficacy of gene transfer for Leber's Congenital Amaurosis". New England Journal of Medicine. 358 (21): 2240–2248. doi:10.1056/NEJMoa0802315. PMC 2829748. PMID 18441370.

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