Cecilia Rosales

发表

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Pratima Chowdary, Savita Rangarajan, Cecilia Rosales, 2011, The New England journal of medicine.

The dynamic DNA methylomes of double-stranded DNA viruses associated with human cancer.

A. Valencia, E. Kieff, M. Fraga, 2008, Genome research.

associated with human cancer The dynamic DNA methylomes of double-stranded DNA viruses Material Supplemental

A. Valencia, E. Kieff, M. Fraga, 2009 .

A Systematic Evaluation of Integration Free Reprogramming Methods for Deriving Clinically Relevant Patient Specific Induced Pluripotent Stem (iPS) Cells

Amit C. Nathwani, Cecilia Rosales, Thomas T. Warner, 2013, PloS one.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Michael Recht, Pratima Chowdary, Deepak Raj, 2014, The New England journal of medicine.

Preferential targeting of disseminated liver tumors using a recombinant adeno-associated viral vector.

M. Lythgoe, Roger Williams, A. Davidoff, 2015, Human gene therapy.

Epigenetic Mouse Models

M. Esteller, C. Rosales, Cecilia Rosales, 2012 .

Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector–mediated intrauterine gene transfer in early-gestation fetal macaques

M. Choolani, J. Chan, Manfred Schmidt, 2018, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

M. Choolani, J. Chan, Manfred Schmidt, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

A. Nienhuis, J. Gray, M. Valentine, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

C. Morton, A. Davidoff, E. Tuddenham, 2018, Blood.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

M. Kay, A. Nienhuis, J. Gray, 2014, The New England journal of medicine.

Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease

Rose M. Sheridan, W. Heywood, K. Mills, 2023, Gene Therapy.

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

P. Fagone, A. Nienhuis, O. Christophe, 2013, Blood.

Potential limits of AAV‐based gene therapy with the use of new transgenes expressing factor IX fusion proteins

C. Négrier, A. Dane, A. Nathwani, 2018, Haemophilia : the official journal of the World Federation of Hemophilia.