Journeys of Gratitude

“The day Manny received his cells back was a pretty emotional day for the whole team“ - Erica Esrick, MD, principle clinical trial investigator

“It was part of my mentality, not knowing how long I was going to live.” - Brendan Whittaker, the first person in the U.S. to receive a genetic therapy at Boston Children's Hospital for CGD under a new international trial

“The biggest takeaway was that the gene therapy was highly effective”-Dr. William Pu on early mouse trial results for reversing heart failure of Barth Syndrome


What is gene therapy?

Gene therapy is the process of introducing DNA into cells in an effort to treat or prevent disease. When a gene mutation (a permanent alteration in the DNA sequence) causes a protein to be missing or faulty, gene therapy may be able to restore the function of that protein by replacing the gene, introducing a healthy copy of the gene or by turning off genes that are causing issues.

The new genes are inserted into cells with either a viral or non viral genetically-engineered vector, a "delivery vehicle" carrying the genetic material. Modified viruses with the infectious capability turned off are used as vectors, because of their natural ability to invade the target cell, delivering the new gene.

Grateful for a healthy baby and a guardian angel doctor

“In the end, showing our gratitude to those who helped heal our son’s body was the best thing we could do to heal all of our hearts.” – Amy. R, mother of 1-year-old

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grateful for a health baby
Emily and Isis Kidney Donation

Emily and Isis: Gratitude in the form of a bean

When Emily became a living donor, she had no idea who would receive her kidney, but that didn’t matter. As it turns out, Isis changed her life, too.

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Police save lives every day, just not this way — a liver for Sloan

"We are grateful for every precious moment we have with our daughter, Sloan. Thank you to our family and friends for their endless love, support and prayers. And to Sloan’s living donor, Steven, for this amazing gift of life." - Sloan's mom, Sarah


Read about Sloan’s liver transplant

sloan transplant update

Books that teach gratitude

With Thanksgiving on the horizon, take a moment to curl up with a good book that can help your little ones experience gratitude. Here are a few suggestions, for ages 4 and up.


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