Margaret V. Ragni
University of Pittsburgh
288 Papers
2.3K Citations
Margaret V. Ragni is an academic researcher from University of Pittsburgh. The author has contributed to research in topics: Medicine & Von Willebrand disease. The author has an hindex of 59, co-authored 265 publications.
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Papers
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
Catherine S. Manno,Glenn F. Pierce,Valder R. Arruda,Bertil Glader,Margaret V. Ragni,Rasko Jj,Margareth C. Ozelo,Keith Hoots,Blatt P,Barbara A. Konkle,Michael D. Dake,Robin D. Kaye,Mahmood K. Razavi,A Zajko,James L. Zehnder,Pradip Rustagi,Hiroyuki Nakai,A Chew,Debra G.B. Leonard,Debra G.B. Leonard,J F Wright,Ruth Lessard,Jurg M. Sommer,Michael Tigges,Denise E. Sabatino,A Luk,Haiyan Jiang,Federico Mingozzi,Linda B. Couto,Hildegund C.J. Ertl,Katherine A. High,Katherine A. High,Mark A. Kay +32 more
TL;DR: In this article, a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic levels of F.IX in dogs with severe hemophilia B.
2.1K
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
Mark A. Kay,Catherine S. Manno,Catherine S. Manno,Margaret V. Ragni,Peter J. Larson,Peter J. Larson,Linda B. Couto,Alan McClelland,Bertil Glader,Amy J. Chew,Shing J Tai,Roland W. Herzog,Valder R. Arruda,Fred Johnson,Ciaran Scallan,Erik D. Skarsgard,Alan W. Flake,Alan W. Flake,Katherine A. High,Katherine A. High +19 more
TL;DR: Evidence of gene expression at low doses of vector suggests that dose calculations based on animal data may have overestimated the amount of vector required to achieve therapeutic levels in humans, and that the approach offers the possibility of converting severe haemophilia B to a milder form of the disease.
1.1K
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Catherine S. Manno,Amy J. Chew,Sylvia Hutchison,Peter J. Larson,Roland W. Herzog,Valder R. Arruda,Shing Jen Tai,Margaret V. Ragni,Arthur A. Thompson,Margareth C. Ozelo,Linda B. Couto,Debra G.B. Leonard,Fred Johnson,Alan McClelland,Ciaran Scallan,Erik D. Skarsgard,Alan W. Flake,Mark A. Kay,Katherine A. High,Bertil Glader +19 more
TL;DR: Results of the first parenteral administration of rAAV demonstrate that administration of AAV vector by the intramuscular route is safe at the doses tested and effects gene transfer and expression in humans in a manner similar to that seen in animals.
799
CD8(+) T-cell responses to adeno-associated virus capsid in humans.
Federico Mingozzi,Marcela V. Maus,Marcela V. Maus,Daniel J. Hui,Denise E. Sabatino,Samuel L. Murphy,John E.J. Rasko,John E.J. Rasko,Margaret V. Ragni,Catherine S. Manno,Catherine S. Manno,Jurg M. Sommer,Haiyan Jiang,Glenn Pierce,Hildegund C.J. Ertl,Katherine A. High,Katherine A. High,Katherine A. High +17 more
TL;DR: It is shown that healthy subjects carry AAV capsid–specific CD8+ T cells and that AAV-mediated gene transfer results in their expansion, and that no such expansion occurs in mice after A AV- mediated gene transfer.
730
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
Lindsey A. George,Spencer K. Sullivan,Adam Giermasz,John E.J. Rasko,John E.J. Rasko,Benjamin J. Samelson-Jones,Jonathan M. Ducore,Adam Cuker,Lisa M. Sullivan,Suvankar Majumdar,Jerome M. Teitel,Catherine E. McGuinn,Margaret V. Ragni,Alvin Luk,Daniel Hui,J. Fraser Wright,Yifeng Chen,Yun Liu,Katie Wachtel,Angela Winters,Stefan Tiefenbacher,Valder R. Arruda,Johannes C.M. Van der Loo,Olga Zelenaia,Daniel Takefman,Marcus E. Carr,Linda B. Couto,Xavier M. Anguela,Katherine A. High +28 more
TL;DR: Transgene‐derived factor IX coagulant activity enabled the termination of baseline prophylaxis and the near elimination of bleeding and factor use in 10 participants with hemophilia who received the same vectors.
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