[HTML][HTML] EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity
…, B Neven, A Schulz, M Slatter, P Veys… - Bone Marrow …, 2021 - nature.com
Inborn errors of immunity (IEI) are a group of rare heterogeneous diseases. Currently, more
than 400 monogenetic IEI have been identified and increasingly a genetic diagnosis can be …
than 400 monogenetic IEI have been identified and increasingly a genetic diagnosis can be …
[HTML][HTML] Wiskott-Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment
HD Ochs, AH Filipovich, P Veys, MJ Cowan… - Biology of Blood and …, 2009 - Elsevier
… In contrast, patients with lymphocytes not expressing WASP or expressing only truncated
WASP are more likely to have the classic WAS phenotype (P < .001). Note that progression to a …
WASP are more likely to have the classic WAS phenotype (P < .001). Note that progression to a …
[HTML][HTML] Chronic granulomatous disease: lessons from a rare disorder
BH Segal, P Veys, H Malech, MJ Cowan - Biology of Blood and Marrow …, 2011 - Elsevier
Chronic granulomatous disease (CGD) is a rare primary immunodeficiency with X-linked or
autosomal recessive inheritance involving defects in genes encoding phox proteins, which …
autosomal recessive inheritance involving defects in genes encoding phox proteins, which …
Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
Autologous T cells engineered to express chimeric antigen receptor against the B cell antigen
CD19 (CAR19) are achieving marked leukemic remissions in early-phase trials but can be …
CD19 (CAR19) are achieving marked leukemic remissions in early-phase trials but can be …
Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99
…, S Müller, A Cant, M Cavazzana-Calvo, P Veys… - The Lancet, 2003 - thelancet.com
… Significant improvements have occurred over time in survival after both HLA-identical (p=0·04)
and nonidentical (p=0·0007) stem-cell transplantation (figure 1). SCID phenotype also …
and nonidentical (p=0·0007) stem-cell transplantation (figure 1). SCID phenotype also …
Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?
…, L Grandin, P Taupin, AJ Cant, P Veys… - Journal of allergy and …, 2010 - Elsevier
BACKGROUND: Hematopoietic stem cell transplantation remains the only treatment for
most patients with severe combined immunodeficiencies (SCIDs) or other primary …
most patients with severe combined immunodeficiencies (SCIDs) or other primary …
Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL treated with a low-affinity CD19 CAR
…, S Samarasinghe, N Goulden, A Vora, P Veys… - Nature medicine, 2019 - nature.com
… b, Burden of F-Luc + tumor cells was quantified and measured as maximum photon per s
per cm 2 per steradian (p per s per cm 2 per sr). Lines represent cumulative results of light …
per cm 2 per steradian (p per s per cm 2 per sr). Lines represent cumulative results of light …
[HTML][HTML] Indications for autologous and allogeneic hematopoietic cell transplantation: guidelines from the American Society for Blood and Marrow Transplantation
Approximately 20,000 hematopoietic cell transplantation (HCT) procedures are performed
in the United States annually. With advances in transplantation technology and supportive …
in the United States annually. With advances in transplantation technology and supportive …
[HTML][HTML] CAR T cells with dual targeting of CD19 and CD22 in pediatric and young adult patients with relapsed or refractory B cell acute lymphoblastic leukemia: a …
…, R Hough, S Ghorashian, A Vora, D Bonney, P Veys… - Nature medicine, 2021 - nature.com
Chimeric antigen receptor (CAR) T cells targeting CD19 or CD22 have shown remarkable
activity in B cell acute lymphoblastic leukemia (B-ALL). The major cause of treatment failure is …
activity in B cell acute lymphoblastic leukemia (B-ALL). The major cause of treatment failure is …
Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two …
Background Genome-edited donor-derived allogeneic anti-CD19 chimeric antigen receptor
(CAR) T cells offer a novel form of CAR-T-cell product that is available for immediate clinical …
(CAR) T cells offer a novel form of CAR-T-cell product that is available for immediate clinical …