one of my colleagues duritn my orthopedic residency had this condition and it caused him to drop out of the program since the stress and hours were too much for his body to handle and started getting respiratory issues. The issue of correcting the point mutation on the DNA that causes Cystic fibrosis was one that has been project by many gene therapy labs for over 30 years. Yet no cure. The genes are now known to be difficult if not impossible to manipulate in humans. So the answer may be stem cell therapy, but since your friend has a bad copy of the genes, she would need fetal cells and that is not allowed in the USA and man never be allowed. So the best treatment is a good pulmonologist, medications as needed and for your girlfriend to stay as healthy as possible.

This is a good question. When people are diagnosed with an ailment, it is typical to experience emotions from denial, anger, hopelessness, acceptance, and others. People often lose faith in traditional medicine and sometimes even seek homeopathic remedies. Cystic fibrosis is not my area, but I did study molecular genetics in college and pondered genetic therapy for other diseases. This is a very difficult question to answer. There are countless laboratories and scientists studying therapeutic options for cystic fibrosis throughout the world.

https://www.cff.org/Our-Research/Our-Research-Approach/

One of the reasons that restriction enzymes are not used to repair human genes is that these enzymes must be introduced into cells that live in the patient. As you may know, the enzymes would be required to affect only the cells and genes that are responsible for the disease. One specific difficulty is the delivery of the enzyme specifically into the affected cells. This can sometimes be done using a "viral vector". This means that a cold virus, for instance, can be genetically engineered to deliver genetic material into cells. However, genetic engineering and selective "in vivo" cell infection are theoretically possible. However genetic errors lead to mutations. And a genetic mutation is what led to cystic fibrosis.

There have, indeed been attempts at gene therapy with cystic fibrosis. However, it appears that improvement has thus far been modest. To learn more about this, I would recommend a PubMed website search. This is far more potent than a Google search for medical issues.

I found this article. It details a gene therapy study that demonstrated modest improvement in CF patients:

Repeated nebulisation of non-viral CFTR gene therapy in

patients with cystic fi brosis: a randomised, double-blind,

placebo-controlled, phase 2b trial

A good answer to your question is beyond the scope of this forum. And this is a classic example of how many folks who do "internet" research can be misled into thinking "theoretical" procedures or processes can be "clinically" applicable to every day patient care--particularly in genetics. There is a big difference between "possible", "practical", and "in everyday use". Sometimes it represents decades between them.

I respect your affinity and care for your Girl friend. Genetic science is evolving and many researchers are using tremendous amount of available resources to use Gene alteration treatments. However, research on Gene alterations remains to be infancy. Realize, that though genetic makeup is possible to alter the genetic map as an ongoing result is very challenging. You hope that soon enough your Girl friend will find the right medical management.

Avi Deshmukh, MD, FACS, MBA, MHA

I am sorry to hear about your girlfriend's health problems. Please continue to be a good support for her.

Please understand that medicine is an evolving science. We may now have an idea that we can manipulate specific genes, and we've got some preliminary results. However, we don't yet know how to specifically and successfully cure your girlfriend's cystic fibrosis. That is a much larger task and these things will be researched as our knowledge and abilities grow. Consider the guy who first built a Conestoga wagon--a very useful and important vehicle. Now ask him why he didn't build a Ferrari with self-driving capabilities. He could make something move that transported people, but he wouldn't have near the knowledge needed to complete that Ferrari, let alone the tools required to build such a complex car.

However, there are many advances in the treatment of CF, and your girlfriend will benefit from these various options.

I can't answer anything different than the reply you got from my previous answers.

At this point in time Gene Therapy is not recognized by the FDA as an approved treatment for CF. Still in the experimental phase. If scientists continue to see results in CF patients it would most likely be recognized as approved treatment, but that could be years from now. All the best to both of you.

This is called gene therapy but still this confirmed to lab level.

This is a great question. If I had the answer to it, I might win a Nobel prize. This is called Gene therapy and while many advances have been made over the years, we're still not quite there. Best of luck you you and your girlfriend.