C. Rosales

发表

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.

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

P. Fagone, A. Nienhuis, O. Christophe, 2014 .

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.

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.

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

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

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.

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

M. Kay, A. Nienhuis, J. Gray, 2010 .

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.

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.

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

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

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

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

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.

Epigenetic Mouse Models

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

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

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

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

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

Rocky Mountain Spotted Fever, A Reemerging Disease in Arizona and Sonora- Case Study

E. Zuckerman, R. Sepúlveda, C. Rosales, 2014 .