0
We're unable to sign you in at this time. Please try again in a few minutes.
Retry
We were able to sign you in, but your subscription(s) could not be found. Please try again in a few minutes.
Retry
There may be a problem with your account. Please contact the AMA Service Center to resolve this issue.
Contact the AMA Service Center:
Telephone: 1 (800) 262-2350 or 1 (312) 670-7827  *   Email: subscriptions@jamanetwork.com
Error Message ......
Editorial |

An Emerging Era of Clinical Benefit From Gene Therapy

Harry L. Malech, MD1; Hans D. Ochs, MD2
[+] Author Affiliations
1Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland
2Department of Pediatrics, University of Washington and Seattle Children’s Research Institute, Seattle
JAMA. 2015;313(15):1522-1523. doi:10.1001/jama.2015.2055.
Text Size: A A A
Published online

Extract

The report in this issue of JAMA by Hacein-Bey Abina and colleagues1 provides strong evidence that gene therapy using myeloid/lymphoid conditioning combined with subsequent infusion of lentivector-transduced autologous CD34+ hematopoietic stem cells (HSCs) achieves substantial restoration of immune function associated with prolonged clinical benefit to patients with severe phenotype Wiskott-Aldrich syndrome (WAS). Prior to treatment, 6 of the 7 patients included in the study had the highest WAS severity disease score of 5; all patients had experienced serious clinical events and complications that are the hallmark of classic WAS, including recurrent viral or bacterial infections, bleeding due to microthrombocytopenia, severe eczema, vasculitis, enteropathy, and other autoimmune problems.

Sign in

Purchase Options

• Buy this article
• Subscribe to the journal
• Rent this article ?

First Page Preview

View Large
First page PDF preview

Figures

Tables

References

CME
Also Meets CME requirements for:
Browse CME for all U.S. States
Accreditation Information
The American Medical Association is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. The AMA designates this journal-based CME activity for a maximum of 1 AMA PRA Category 1 CreditTM per course. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Physicians who complete the CME course and score at least 80% correct on the quiz are eligible for AMA PRA Category 1 CreditTM.
Note: You must get at least of the answers correct to pass this quiz.
Please click the checkbox indicating that you have read the full article in order to submit your answers.
Your answers have been saved for later.
You have not filled in all the answers to complete this quiz
The following questions were not answered:
Sorry, you have unsuccessfully completed this CME quiz with a score of
The following questions were not answered correctly:
Commitment to Change (optional):
Indicate what change(s) you will implement in your practice, if any, based on this CME course.
Your quiz results:
The filled radio buttons indicate your responses. The preferred responses are highlighted
For CME Course: A Proposed Model for Initial Assessment and Management of Acute Heart Failure Syndromes
Indicate what changes(s) you will implement in your practice, if any, based on this CME course.

Multimedia

Some tools below are only available to our subscribers or users with an online account.

1,520 Views
1 Citations
×

Sign in

Purchase Options

• Buy this article
• Subscribe to the journal
• Rent this article ?

Related Content

Customize your page view by dragging & repositioning the boxes below.

See Also...
Articles Related By Topic
Related Collections
PubMed Articles
Jobs
brightcove.createExperiences();