New nanoparticles can perform gene editing in the lungs

Re:New nanoparticles can perform gene editing in the lungs by gfodor on Thursday April 27, 2023 @05:00PM (#35734515)

As someone with CF, I remember hearing about gene therapy as a kid as far back as the late 80s. Thankfully, we didn’t have to wait for it for many CF patients to have their respiratory issues much improved or even resolved with CFTR modulators.

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Re:New nanoparticles can perform gene editing in the lungs by tomcam on Thursday April 27, 2023 @06:40PM (#35735416)

I had no clue. Are you one of the lucky ones?

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Re:New nanoparticles can perform gene editing in the lungs by gfodor on Friday April 28, 2023 @01:12AM (#35737662)

yeah it worked for me. CF is now basically a bad dream.

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Re:New nanoparticles can perform gene editing in the lungs by tomcam on Friday April 28, 2023 @07:50AM (#35740134)

So happy to hear this.

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Re:New nanoparticles can perform gene editing in the lungs by DoreenMichele on Thursday April 27, 2023 @10:47PM (#35736971)

CFTR modulators (a class of drugs to treat CF):

https://www.cff.org/managing-cf/cftr-modulator-therapies

Last I heard, they are life changing for some people with CF. On the downside, they are typically extremely expensive (like $250k annually) and have to be taken for life and don't work for everyone. It depends on your specific CFTR defect, the CFTR being the defective cell channel at the root of all CF symptoms and pathology.

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Re:New nanoparticles can perform gene editing in the lungs by DLTADragonHawk on Thursday April 27, 2023 @11:14PM (#35737099)

I have dropped all medical treatments except for pancreatic enzymes due to these. My pulmonary function tests came back as 104 after over a year of dropping the primary treatments for this being albuteral, "vest", and sinus surgeries. If you qualify for this medication it's life changing.

Medication for those interested. https://www.trikafta.com

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Re:New nanoparticles can perform gene editing in the lungs by eutectic on Friday April 28, 2023 @01:27AM (#35737748)

Why would small molecule drugs like these be so expensive? Limited market?

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Re:New nanoparticles can perform gene editing in the lungs by DoreenMichele on Friday April 28, 2023 @01:46AM (#35737866)

Limited market is likely a factor, yes.

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Re:New nanoparticles can perform gene editing in the lungs by DLTADragonHawk on Friday April 28, 2023 @08:14AM (#35740355)

They are also brand new. For the drug as well the company is actual very helpful and realistically after insurance and the companies assistance I have paid 2, $15 co-pays the last 2 years.

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