X-SCID

Treatment of X-linked Severe Combined Immunodeficiency (X-SCID) by the in utero transplantation of paternal bone marrow.

Fetal Stem Cell Transplantation, In Utero Stemm Cell Publications 2010

Both this child and another who had developed a similar condition in August 2002 had been successfully treated by gene therapy for X-linked severe combined immunodeficiency disease (X-SCID), also known as "bubble baby syndrome."

Gene Therapy Novel Therapeutic Branch of Modern Medicine 2008

The London hospital said 18-month-old Rhys Evans had been cured of the fatal genetic condition X-linked Severe Combined Immunodeficiency Disease X-SCID, commonly known as “baby in the bubble” syndrome.

‘Bubble Boy’ Cured in Gene Therapy Breakthrough | Impact Lab 2002

Differential counts of WBCs showed a dramatic decrease in leukocytes in the X-SCID rats (Table S5).

PLoS ONE Alerts: New Articles Tomoji Mashimo et al. 2010

Table S3 to characterize the immunodeficient phenotypes of the X-SCID rats.

PLoS ONE Alerts: New Articles Tomoji Mashimo et al. 2010

The thymus of the X-SCID rat was severely hypoplastic and consisted of an epithelial cell sheet.

PLoS ONE Alerts: New Articles Tomoji Mashimo et al. 2010

These immunodeficient phenotypes of the X-SCID rats were very similar to those of the previously reported X-SCID mice and were characterized by a nearly complete lack of T-cells,

PLoS ONE Alerts: New Articles Tomoji Mashimo et al. 2010

Differential counts of the white blood cells of Il2rg-deficient (X-SCID) rats.

PLoS ONE Alerts: New Articles Tomoji Mashimo et al. 2010

A major motivation for performing this experiment was the observation that although SCID mouse animal models are the most commonly used in research on drug development, an X-SCID immunodeficient rat model would complement mouse models through the additional advantage of being employed for testing the pharmacodynamics and toxicity of potential therapeutic compounds.

PLoS ONE Alerts: New Articles Tomoji Mashimo et al. 2010

In addition, the X-SCID rats that were established in this study will be valuable in vivo tools for evaluating drug treatment or gene therapy as well as model systems for examining the treatment of xenotransplanted malignancies.

PLoS ONE Alerts: New Articles Tomoji Mashimo et al. 2010