YOU’RE ABOUT TO MEET A YOUNG MAN
PREPARED TO DO WHATEVER IT
TAKES.
TERRY MORGAN IS 24.
HE IS SUFFERING FROM A RARE FORM
OF MUSCULAR DYSTROPHY,
RESTRICTING HIS GROWTH FROM AN
EARLY AGE.
>> MY MOM USED TO GO TO SCHOOL
WITH ME.
I DIDN’T LIKE TO DO STUFF LIKE
THAT.
I GUESS, LIKE, I CAN THROW A
BASEBALL.
>> Reporter: THREATENING HIM
WITH AN EARLY DEATH.
>> THIS IS A TOUGH DISEASE.
IT IS A FORGOTTEN DISEASE.
SOMETIMES YOU FEEL LIKE YOU’RE
ALL BY YOURSELF FIGHTING IT.
>> Reporter: AND FORCING HIS
FAMILY TO LIVE WITH YEARS OF
WORRY.
>> EVERY YEAR, AS TERRY GETS
OLDER, THAT FEAR AND THAT KNOT
IN YOUR STOMACH BECOMES
STRONGER.
I DON’T WANT TO LOSE HIM.
MY KIDS ARE THE REASON I GET UP
IN THE MORNING.
>> KIND OF JUST DEAL WITH IT,
AND LIFE GOES ON.
YOU CAN’T WORRY ABOUT IT EVERY
DAY.
>> Reporter: IT’S HARD TO LISTEN
TO THAT.
>> IMPACTS EVERY FACET OF YOUR
LIFE.
AND IT IS VERY PERSONAL.
AS A KID, IT MAKES YOU FEEL VERY
POWERLESS. IT MAKES YOU FEEL VERY HOPELESS.
IT MAKES YOU FEEL VERY HOPELESS.
>> Reporter: BERTERRY’S BROTHER,
RICHARD, A HARVARD GAJRADUATE,
MADE IT HIS WORK TO GIVE TERRY
AND OTHER PATIENTS WITH RARE
DISEASES HOPE.
>> YOU FIGHT THE BATTLE YOU’RE
MEANT TO FIGHT, AND YOU DON’T
GIVE UP.
>> Reporter: DON’T GIVE UP.
MORGAN CREATED A NON-PROFIT MOWN
FOUNDATION WITH A NAME OF HIS
WISH, PURE DISEASE.
>> THE PACE OF INNOVATION, I
THINK, IS TOO SLOW.
IT IS NOT RIGHT THAT TODAY’S
PATIENTS DON’T GET TO SEE
TOMORROW’S CURES.
IT IS A SCARY SORT OF BATTLE TO
JUMP INTO.
>> Reporter: HE DIDN’T JUMP
ALONE.
HE FOUND A GENETICIST AT YALE
WHO ALSO SUFFERS FROM MUSCULAR
DYSTROPHY.
TOGETHER WITH HIS WIFE, ANGELA,
HE SPEAR-HEADED RESEARCH INTO A
CURE, WITH A GENE EDITING
TECHNIQUE CALLED CRISPA.
>> COULD BE A BREAKTHROUGH
BECAUSE OF CRISPA APPROACH WE’RE
USING.
>> Reporter: WHAT IS IT?
IT IS A REVOLUTION FROM THE LAB
THAT COULD CHANGE LIFE AS WE
KNOW IT.
>> THE CELLS HAVE TAKEN THE DNA
OF INTEREST WHICH, IN THIS CASE,
CARRIES THE CRISPA COMPONENTS.
>> Reporter: YOU CAN CHANGE THE
WORLD IN THIS?
>> THEORETICALLY, YES,
ABSOLUTELY.
>> Reporter: ACTING LIKE
MOLECULAR SCISSORS, IT CAN SNIP
A FAULTY JEAN INGENE IN A STRAND
ALLOWING A REWRITING OF THE
GENETIC CODE.
PROMISING A SOLUTION TO
INCURABLE DISEASES AND INHA
INHABILITATING LIFESTYLE.
>> ONCE TECHNOLOGY EXISTS, IT
EXISTS.
IT TENDS TO ONLY GET BETTER.
>> Reporter: IT IS A UNIQUE WAY
TO ADDRESS UNTREATABLE GENETIC
CONDITIONS INDIVIDUALLY.
RICH IS DETERMINED TO DO THINGS
ACCORDING TO THE BOOK, ONLY
FASTER.
IS IT A RACE AGAINST TIME?
>> ABSOLUTELY.
WE’RE BRINGING TOGETHER THIS
WHOLE TEAM TO PROVIDE CHECKS AND
BALANCES.
DO THE RESEARCH, TALK TO FDA,
THEN DO SOME MORE RESEARCH, THEN
WE GO FOR ARRIVAL.
>> Reporter: IF THE FDA APPROVAL
FOR THE TRIAL COMES THROUGH,
TERRY WILL BE HIS OWN GUINEA PIG
LATER THIS YEAR.
>> THERE WAS REALLY NOTHING OUT
THERE FOR US, FOR ME.
NOW, RICHARD IS WORKING ON, YOU
KNOW, FINDING A CURE, YOU KNOW.
WE HAVE A LITTLE LIGHT AT THE
END OF THE TUNNEL.
TWO YEARS AGO, IT WAS ALL BLACK.
>> I HOPE THAT TERRY PAVES THE
WAY FOR OTHERS.
AND THAT NO FAMILY HAS TO SUFFER
AGAIN.
>> Reporter: TERRY SAYS HE HAS
NOTHING TO LOSE.
A LIFE TO WIN.
AND THIS TREATMENT COULD HOLD
OUT HOPE FOR PEOPLE WITH MANY DIFFERENT CONDITIONS.
DIFFERENT CONDITIONS.
SICKLE CELL AMEANNEMIA, FOR EXAM
IT IS NOT WITHOUT CONTROVERSY,
BUT IT IS HOPE.
>> I’M TOUCHED BY HIS BROTHER
MAKING IT HIS LIFE’S MISSION.
WHAT RISKS DOES THIS NEW SCIENCE
AND TRIAL INVOLVE?
YOU TALKED ABOUT SOME OF THE
ETHICAL IMPLICATIONS.
WHAT ABOUT THE RISKS?
>> Reporter: YEAH.
I MEAN, YOU KNOW, THERE IS A
DANGER FOR TERRY, THAT HIS
IMMUNE SYSTEM COULD OVERREACT.
I MEAN, IT IS A LIFE-THREATENING
DANGER.
SO HE IS GOING TO BE A PIONEER.
HE IS GOING TO BE ON THE
FOREFRONT OF THIS, TAKING A RISK
FOR HIMSELF, AS YOU SAW THERE,
BUT ALSO, OF COURSE, FOR MANY
OTHERS.
YOU KNOW, SAVANNAH, YOU LOOK AT
TERRY, AND HE DOESN’T LOOK LIKE
THE STRONGEST GUY.
I SPENT A SHORT TIME WITH HIM,
AND I CAN TELL YOU, HE IS SO