<story><title>‘Bubble Boys’ Cured in Medical Breakthrough Using Gene Therapy</title><url>https://www.bloomberg.com/news/articles/2019-04-17/-bubble-boys-cured-in-medical-breakthrough-using-gene-therapy</url></story><parent_chain></parent_chain><comment><author>scop</author><text>I am an adult living with a primary immune deficiency and this is quite surreal. Dr. Jennifer Puck, on the left in the photo, is my doctor at UCSF. Let me just say that she is an incredible doctor.&lt;p&gt;I’ve had a vague hope in gene therapy for many years, but have never really believed I would live to see it come to market as a real treatment.&lt;p&gt;I’ve told my family many times that “maybe gene therapy will be the fix”. Today is the first day I’ve actually believed those words.</text></comment>
<story><title>‘Bubble Boys’ Cured in Medical Breakthrough Using Gene Therapy</title><url>https://www.bloomberg.com/news/articles/2019-04-17/-bubble-boys-cured-in-medical-breakthrough-using-gene-therapy</url></story><parent_chain></parent_chain><comment><author>rgejman</author><text>The actual study is here. It&amp;#x27;s a remarkable achievement: &lt;a href=&quot;https:&amp;#x2F;&amp;#x2F;www.nejm.org&amp;#x2F;doi&amp;#x2F;full&amp;#x2F;10.1056&amp;#x2F;NEJMoa1815408&quot; rel=&quot;nofollow&quot;&gt;https:&amp;#x2F;&amp;#x2F;www.nejm.org&amp;#x2F;doi&amp;#x2F;full&amp;#x2F;10.1056&amp;#x2F;NEJMoa1815408&lt;/a&gt;&lt;p&gt;No CRISPR&amp;#x2F;Cas9 was used in this study.&lt;p&gt;We&amp;#x27;ll have to wait and see if any of the patients develop hematologic malignancies (blood cancers).&lt;p&gt;I predict we&amp;#x27;ll see dozens of papers like this in the next few years. Sickle cell and thalessemias are low hanging fruit.</text></comment>