NEWS

The SSADH Association has granted the funding request from Dr. Henry Lee and Dr. Alex Rotenberg of Boston Children’s Hospital (BCH) / Harvard Medical School seeking nine month interim funding of $48,990 to keep the gene replacement therapy project on the SSADH inducible mouse model moving forward until their next funding is secured from outside sources.

Prior to Dr. Lee’s gene replacement therapy intervention, BCH had been unable to keep the SSADH colony of mice alive for more than 29 days.  This mortality threshold was also consistent with the mice colony that was under the care of Dr. Mike Gibson at Washington State University for over fifteen years. With Dr. Lee’s intervention techniques, mice were able to survive beyond 29 days indicating some initial success with the project.  In recent correspondence however, Dr. Lee shared with us that some of the treated mice had not survived beyond 49 days which requires further evaluation into the various restoration parameters in the inducible mouse model of SSADH Deficiency.  The primary purpose of this funding request is to examine those parameters.

Although gene replacement therapy had doubled the life span of the SSADH affected mice, it was anticipated that the mice would survive beyond 49 days because the treated mice’s growth rate had caught up to the unaffected mice and they were exhibiting normal behavior (for mice). 

Dr. Lee is expecting additional external funding to continue his work with gene replacement therapy on the SSADH inducible mouse model progressing towards a clinical trial and potential treatment option for SSADH patients.  Dr. Lee’s grant from the SSADH Association will provide funding from April 1, 2023 thru December of 2023.