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ESI Special Topics, September 2007
Citing URL: http://www.esi-topics.com/fmf/2007/september07-MarinaCavazzanaCalvo.html

From •>>SEPTEMBER 2007

Marina Cavazzana-Calvo answers a few questions about this September's fast moving front in the field of Biology & Biochemistry. 

Field: Biology & Biochemistry
Article: Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
Authors: Hacein-Bey-Abina, S;Le Deist, F;Carlier, F;Bouneaud, C;Hue, C;De Villartay, J;Thrasher, AJ;Wulffraat, N;Sorensen, R;Dupuis-Girod, S;Fischer, A;Cavazzana-Calvo, M;Davies, EG;Kuis, W;Lundlaan, WHK;Leiva, L
Journal: N ENGL J MED, 346 (16): 1185-1193, APR 18 2002
Addresses:
Hop Necker Enfants Malad, Lab Therapie Cellulaire & Gen, 149 Rue Sevres, F-75015 Paris, France.
Hop Necker Enfants Malad, Lab Therapie Cellulaire & Gen, F-75015 Paris, France.
Hop Necker Enfants Malad, Lab INSERM, Paris, France.
Hop Necker Enfants Malad, Lab Immunol Pediat, Paris, France.
Hop Necker Enfants Malad, Unite Immunol & Hematol Pediat, Paris, France.
Inst Pasteur, Unite Biol Gene, Paris, France.
Inst Child Hlth, Mol Immunol Unit, London, England.
Wilhelmina Kinderziekenhuis Lundlaan, Dept Immunol & Hematol, Utrecht, Netherlands.
Louisiana State Univ, Hlth Sci Ctr, Dept Pediat, New Orleans, LA USA.
Great Ormond St Hosp Children, Natl Hlth Serv Trust, London, England.


   Why do you think your paper is highly cited?

Because it reports on the follow-up of the first patients who restored a normal phenotype after an ex vivo gene therapy trial.

   Does it describe a new discovery, methodology, or synthesis of knowledge?

It’s a synthesis of knowledge in molecular medicine.

   How did you become involved in this research and were there any particular problems encountered along the way?

Because I am greatly interested in the treatment of patients affected by inherited disorders of the hematopoietic system who do not have an HLA-compatible donor leading us to treat them. Therefore, the use of autologous gene-modified stem cells represents an extremely powerful strategy.

   Where do you see your research leading in the future?

To genome surgery involving target genes of therapeutic interest, leading to correction of some of these diseases by homologous recombination.End

Marina Cavazzana-Calvo, Pr. in Hematology, M.D. Ph.D
Director of the Biotherapy Dept -Hopital Necker -Enfants Malades
Paris, France
 Website

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ESI Special Topics, September 2007
Citing URL: http://www.esi-topics.com/fmf/2007/september07-MarinaCavazzanaCalvo.html
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