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1 . Academic Journal
Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a
저자
by
George, Lindsey A.
;
Ragni, Margaret V.
;
Samelson-Jones, Ben J.
;
Cuker, Adam
;
Runoski, Alexa R.
, et al.
소스
In
Blood
8 December 2017 130 Supplement 1:604-604
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7 . Academic Journal
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
저자
by
George, Lindsey A.
;
Sullivan, Spencer K.
;
Giermasz, Adam
;
Rasko, John E.J.
;
Samelson-Jones, Benjamin J.
, et al.
소스
The New England Journal of Medicine
. Dec 07, 2017 377(23):2215-2227
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7 . Academic Journal
A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds
저자
by
High, Katherine A.
;
George, Lindsey A.
;
Eyster, M. Elaine
;
Sullivan, Spencer K.
;
Ragni, Margaret V.
, et al.
소스
BLOOD
; NOV 29 2018, 132 3p. Supplement: 1
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7 . Academic Journal
Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of > 30% without Immunosuppression
저자
by
George, Lindsey A.
;
Sullivan, Spencer K.
;
Giermasz, Adam
;
Ducore, Jonathan M.
;
Teitel, Jerome M.
, et al.
소스
BLOOD
; DEC 2 2016, 128 22, 7p.
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7 . Academic Journal
The State of Gene Therapies: The FDA Perspective.
저자
by
Takefman, Daniel
;
Bryan, Wilson
.
소스
Molecular Therapy
. May2012, Vol. 20 Issue 5, p877-878. 2p.
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4 . Periodical
Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression
저자
by
George, Lindsey A.
;
Sullivan, Spencer K.
;
Giermasz, Adam
;
Ducore, Jonathan M.
;
Teitel, Jerome M.
, et al.
소스
Blood; December 2016, Vol. 128 Issue: 22 p3-3, 1p
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7 . Academic Journal
SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B at the Low Vector Dose Achieved Sustained, Continuous Factor IX Activity Levels Adequate for Endogenous Prophylaxis Preventing Bleeding Episodes and Reducing the Risk of Immune Response
저자
by
George, Lindsey A.
;
Sullivan, Spencer K.
;
Giermasz, Adam
;
Ducore, Jonathan
;
Rasko, John
, et al.
소스
MOLECULAR THERAPY
; MAY 2017, 25 5, p331-p332, 2p. Supplement: 1
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7 . Academic Journal
SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - Sustained Fix Activity, Persistent Endogenous Prophylaxis and Improved Quality of Life at One Year and Beyond
저자
by
George, Lindsey A.
;
Sullivan, Spencer K.
;
Giermasz, Adam
;
Samelson-Jones, Ben J.
;
Ducore, Jonathan M.
, et al.
소스
MOLECULAR THERAPY
; MAY 2018, 26 5, p1-p1, 1p. Supplement: 1
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7 . Academic Journal
SPK-8011: Preliminary Results from a Phase 1/2 Trial of Investigational Gene Therapy for Hemophilia Confirm Transgene Derived Increases in FVIII Activity That Are Persistent and Stable beyond Eight Months
저자
by
Sullivan, Spencer K.
;
George, Lindsey A.
;
Ragni, Margaret V.
;
Samelson-Jones, Ben
;
Cuker, Adam
, et al.
소스
MOLECULAR THERAPY
; MAY 2018, 26 5, p163-p163, 1p. Supplement: 1
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