Gene Therapy cures sickle cell in teenage boy

[ScienceRocks]

Gene Therapy cures sickle cell in teenage boy

A French teenager's sickle cell disease has been reversed using a pioneering gene therapy treatment. The world-first procedure at Necker Children's Hospital in Paris offers hope to millions of people with the blood disorder.

Doctors removed his bone marrow - the part of the body that makes blood. They then genetically altered it in a lab to compensate for the defect in his DNA that caused the disease. The altered bone marrow has been making healthy red blood cells for 15 months. He is no longer on any medication.

Next Big Future: Gene Therapy cures sickle cell in teenage boy

Gene Therapy is starting to help people!

 

[irosie91]

thanks matt------would that treatment be a one time thing?-------the repaired bone marrow would be a FOREVER thing?

 

[ScienceRocks]

thanks matt------would that treatment be a one time thing?-------the repaired bone marrow would be a FOREVER thing?

I believe so.

 

[Fenton Lum]

Cyctis Fibrosis is another disease that could potentially benefit from this approach as well. We were working on this back in the 1990's using adeno associated viral vetors to deliver copies of the corrected gene sequence to CF patients. 90-95% of the human population is infected with adenovirus but it is nonpathogenic in humans. The approach was to gentically engineer the corrected cystic fibrosis transmembrane conductance regulator (CFTR) protein sequence in between two terminal inverted repeat sequences, which were also engineered into the vector. The inverted terminal repeat sequences flanking the corected CFTR sequence allowed insertion of the corrected CFTR into patient chromosomal DNA. The issue at the time was the size of the CFTR sequence; it was at the upper limit range of what could successfully get inserted into chromosomal DNA using this vector (~4.5KB if I recall correctly).

Since adeno associated virus is nonpathogenic in humans and since the tissue targetted for this medical infection of these viral vectors was lung tissue, the administration could be accomplished via a nasal spray. Worked in cell culture, but at that time we could never get high enough viral vector infection rates into enough patient lung cells, and enough copies of the corrected CFTR gene sequence into chromosomal DNA to achieve enough corrected CFTR function.

 

[irosie91]

Cyctis Fibrosis is another disease that could potentially benefit from this approach as well. We were working on this back in the 1990's using adeno associated viral vetors to deliver copies of the corrected gene sequence to CF patients. 90-95% of the human population is infected with adenovirus but it is nonpathogenic in humans. By gentically engineering the corrected cystic fibrosis transmembrane conductance regulator (CFTR) protein sequence in between two terminal inverted repeat sequences also engineered into the vector. The inverted repeat sequences allowed insertion into patient chromosomal DNA. The issue at the time was the size of the CFTR sequence; it was at the upper limit range of what could successfully get inserted into chromosomal DNA using this vector (~4.5KB if I recall correctly).

Since adeno associated virus is nonpathogenic in humans and since the tissue targetted for this medical infection of these viral vectors was lung tissue, the administration could be accomplished via a nasal spray. Worked in cell culture, but at that time we could never get high enough viral vector infection rates into enough patient lung cells and enough copies of the corrected gene sequence into chromosomal DNA to achieve enough corrected CFTR function.

I AM IMPRESSED------maybe someday------maybe it will be a long term repetitive thing-------starting young????

 

[Fenton Lum]

thanks matt------would that treatment be a one time thing?-------the repaired bone marrow would be a FOREVER thing?

Hard to tell from the links and only an abstract.

Hematopoietic stem cell transplantation - Wikipedia

 

[Fenton Lum]

Cyctis Fibrosis is another disease that could potentially benefit from this approach as well. We were working on this back in the 1990's using adeno associated viral vetors to deliver copies of the corrected gene sequence to CF patients. 90-95% of the human population is infected with adenovirus but it is nonpathogenic in humans. By gentically engineering the corrected cystic fibrosis transmembrane conductance regulator (CFTR) protein sequence in between two terminal inverted repeat sequences also engineered into the vector. The inverted repeat sequences allowed insertion into patient chromosomal DNA. The issue at the time was the size of the CFTR sequence; it was at the upper limit range of what could successfully get inserted into chromosomal DNA using this vector (~4.5KB if I recall correctly).

Since adeno associated virus is nonpathogenic in humans and since the tissue targetted for this medical infection of these viral vectors was lung tissue, the administration could be accomplished via a nasal spray. Worked in cell culture, but at that time we could never get high enough viral vector infection rates into enough patient lung cells and enough copies of the corrected gene sequence into chromosomal DNA to achieve enough corrected CFTR function.

I AM IMPRESSED------maybe someday------maybe it will be a long term repetitive thing-------starting young????

The beauty of that approach toward genetically inherited diseases is, if you can ever get enough corrected gene sequence copies inserted into chromosomal DNA, the “fix” is permanent. If by “starting young” you mean with young patients, theoretically it shouldn’t matter. I’ve been away from that field for some time, but at that time most CF patients didn’t get too far into middle age.

 

[irosie91]

thanks matt------would that treatment be a one time thing?-------the repaired bone marrow would be a FOREVER thing?

Hard to tell from the links and only an abstract.

Hematopoietic stem cell transplantation - Wikipedia

seems terrific-----my question is------FROM WHERE COMETH---
new Hematopoietic stem cells?------are they BORN from some kind
of matrix containing multipotential cells-----or do they snap off of
full blown big guys producing all sorts of stuff like-------platelets and
leukocytes and RBC 's I cannot remember the details of Hematopoiesis
(spelling?) It was a long time ago

 

[Fenton Lum]

thanks matt------would that treatment be a one time thing?-------the repaired bone marrow would be a FOREVER thing?

Hard to tell from the links and only an abstract.

Hematopoietic stem cell transplantation - Wikipedia

seems terrific-----my question is------FROM WHERE COMETH---
new Hematopoietic stem cells?------are they BORN from some kind
of matrix containing multipotential cells-----or do they snap off of
full blown big guys producing all sorts of stuff like-------platelets and
leukocytes and RBC 's I cannot remember the details of Hematopoiesis
(spelling?) It was a long time ago

Shit girl, don't get me to telling lies, I don't recall all that either.

Blood and Bone Marrow Transplant - NHLBI, NIH

And this:

"Bone marrow (stem cell) transplant is the only treatment available today that can cure sickle cell disease. If the transplant is successful, the patient is cured from sickle cell disease."
https://www.stjude.org/content/dam/...m-cell-transplant-for-sickle-cell-disease.pdf

 

[anotherlife]

Gene therapy? Genetically modified crops and genetically modified people? Do you know that aging and death is also a genetic programming? Theoretically you could reprogram the genes continually to reverse aging and to eliminate death. Then imagine that you are my slave not for life but for eternity. Hehehe.

 

[ScienceRocks]

Thankfully anotherlife,

Slavery will remain illegal.