Not ubiquitous but quite personal:
CFTR protein/CFTR gene/small molecule mechanisms for drugs
Ability to call Cystic Fibrosis a chronic/manageable condition
CFTR protein/CFTR gene/small molecule mechanisms for drugs
Ability to call Cystic Fibrosis a chronic/manageable condition
December 5, 2024 at 6:27 AM
Not ubiquitous but quite personal:
CFTR protein/CFTR gene/small molecule mechanisms for drugs
Ability to call Cystic Fibrosis a chronic/manageable condition
CFTR protein/CFTR gene/small molecule mechanisms for drugs
Ability to call Cystic Fibrosis a chronic/manageable condition
Big day for the CF community.
From an incurable genetic condition to a once-day-dose-of-medicine to manage your chronic health condition in the course of a decade
www.cff.org/news/2024-12...
From an incurable genetic condition to a once-day-dose-of-medicine to manage your chronic health condition in the course of a decade
www.cff.org/news/2024-12...
FDA Approves New, Once-a-Day CFTR Modulator for People With CF
A new CFTR modulator treatment called Alyftrek has been approved for people with CF ages 6 and older who have CFTR mutations that are eligible for Trikafta, as well as 31 other rare mutations that hav...
www.cff.org
December 20, 2024 at 11:22 PM
Big day for the CF community.
From an incurable genetic condition to a once-day-dose-of-medicine to manage your chronic health condition in the course of a decade
www.cff.org/news/2024-12...
From an incurable genetic condition to a once-day-dose-of-medicine to manage your chronic health condition in the course of a decade
www.cff.org/news/2024-12...
A great example is the team of organizers & Bangladeshi drug manufacturers working to create generic versions of groundbreaking CFTR inhibitors, so that people living worldwide with CF aren’t priced out or excluded from the US patent-controlled version:
😮A $370,000-per-year cystic fibrosis treatment will cost as little as $2,000 now that a Bangladeshi drug manufacturer will make a generic version.
👏The “revolutionary” drug delivers normal life expectancy to patients, who otherwise die as young adults.
www.theguardian.com/society/2026...
👏The “revolutionary” drug delivers normal life expectancy to patients, who otherwise die as young adults.
www.theguardian.com/society/2026...
‘We were sitting with our calculator saying “we can afford that!”’ Joy for families as cystic fibrosis drug prices fall within reach
The cost of medication was too high for thousands of CF sufferers around the world. Now a Bangladeshi company is making a generic version that will change lives
www.theguardian.com
January 26, 2026 at 12:00 AM
A great example is the team of organizers & Bangladeshi drug manufacturers working to create generic versions of groundbreaking CFTR inhibitors, so that people living worldwide with CF aren’t priced out or excluded from the US patent-controlled version:
Vought's daughter takes Trikafta for cystic fibrosis. The CFTR gene was isolated in 1989 with NIH dollars. Every step of the 30-year pipeline was federally funded academic research.
His OMB is now strangling the exact work that produced the drug keeping her alive.
His OMB is now strangling the exact work that produced the drug keeping her alive.
May 31, 2026 at 12:21 AM
Vought's daughter takes Trikafta for cystic fibrosis. The CFTR gene was isolated in 1989 with NIH dollars. Every step of the 30-year pipeline was federally funded academic research.
His OMB is now strangling the exact work that produced the drug keeping her alive.
His OMB is now strangling the exact work that produced the drug keeping her alive.
My son Jimmy was born with cystic fibrosis in 1986.
The man below is a hero in our lives.
Francis Collins was a key figure in the discovery of the cystic fibrosis gene (CFTR) in 1989.
On 60 minutes tonight. www.axios.com/2025/04/28/t...
The man below is a hero in our lives.
Francis Collins was a key figure in the discovery of the cystic fibrosis gene (CFTR) in 1989.
On 60 minutes tonight. www.axios.com/2025/04/28/t...
Ex-NIH chief says he was "not allowed to speak" under 2nd Trump administration
New NIH director Jay Bhattacharya said at his confirmation hearing he'd "establish a culture of respect for free speech."
www.axios.com
April 28, 2025 at 3:05 AM
My son Jimmy was born with cystic fibrosis in 1986.
The man below is a hero in our lives.
Francis Collins was a key figure in the discovery of the cystic fibrosis gene (CFTR) in 1989.
On 60 minutes tonight. www.axios.com/2025/04/28/t...
The man below is a hero in our lives.
Francis Collins was a key figure in the discovery of the cystic fibrosis gene (CFTR) in 1989.
On 60 minutes tonight. www.axios.com/2025/04/28/t...
Pneumologie:
- CFTR Modulatoren bei Mukoviszidose
- Nintedanib und Pirfenidon bei Lungenfibrose
- Biologika beim schweren Asthma
Bonus: die gesamte Lungenkrebstherapie hat sich bedeutend verändert
- CFTR Modulatoren bei Mukoviszidose
- Nintedanib und Pirfenidon bei Lungenfibrose
- Biologika beim schweren Asthma
Bonus: die gesamte Lungenkrebstherapie hat sich bedeutend verändert
February 14, 2026 at 7:33 AM
Pneumologie:
- CFTR Modulatoren bei Mukoviszidose
- Nintedanib und Pirfenidon bei Lungenfibrose
- Biologika beim schweren Asthma
Bonus: die gesamte Lungenkrebstherapie hat sich bedeutend verändert
- CFTR Modulatoren bei Mukoviszidose
- Nintedanib und Pirfenidon bei Lungenfibrose
- Biologika beim schweren Asthma
Bonus: die gesamte Lungenkrebstherapie hat sich bedeutend verändert
As I recall he is a eugenics proponent and now we know he has a daughter with a genetically transmitted disease that requires both parents to pass on the CFTR gene. CF occurs in all races but is most prominent in white people of Northern European ancestry. Oh the irony...
February 12, 2025 at 5:51 PM
As I recall he is a eugenics proponent and now we know he has a daughter with a genetically transmitted disease that requires both parents to pass on the CFTR gene. CF occurs in all races but is most prominent in white people of Northern European ancestry. Oh the irony...
Before coming to NIH Collins was at U Michigan. In the late 1980s his team identified CFTR as the gene mutated in cystic fibrosis (CF). This was a heroic achievement & paved the way for effective therapy — including the child of a high-ranking OMB official who is currently trying to destroy NIH.
Francis Collins led the mapping of the human genome, and chose to do big scientific to benefit the public. He is a a former NIH Director.
He just resigned his position in government. His resignation letter:
www.nytimes.com/2025/03/01/u...
He just resigned his position in government. His resignation letter:
www.nytimes.com/2025/03/01/u...
March 1, 2025 at 6:56 PM
Before coming to NIH Collins was at U Michigan. In the late 1980s his team identified CFTR as the gene mutated in cystic fibrosis (CF). This was a heroic achievement & paved the way for effective therapy — including the child of a high-ranking OMB official who is currently trying to destroy NIH.
A diagnosis of cystic fibrosis no longer means you are guaranteed to die in your 20s, due to advances in drug therapy like CFTR Modulators
July 14, 2026 at 6:18 AM
A diagnosis of cystic fibrosis no longer means you are guaranteed to die in your 20s, due to advances in drug therapy like CFTR Modulators
CFTR gene cloned in 1989. Combined CFTR modulators elexacaftor-tezacaftor-ivacaftor (ETI) approved in 2019 -->
"The introduction of ETI has dramatically reduced the need for lung transplants among people with CF, reflecting significant improvements in lung function and disease management."🧪⚕️
"The introduction of ETI has dramatically reduced the need for lung transplants among people with CF, reflecting significant improvements in lung function and disease management."🧪⚕️
Impact of elexacaftor-tezacaftor-ivacaftor in lung transplantation for cystic fibrosis in the United States
Cystic fibrosis (CF) is an autosomal recessive condition leading to progressive lung disease and often necessitating lung transplantation. Historically, CF has been one of the leading indications for lung transplants in the United States. The advent of CF transmembrane conductance regulator (CFTR) modulators, particularly elexacaftor-tezacaftor-ivacaftor (ETI), has significantly improved clinical outcomes for people with CF (pwCF), offering potential alterations in disease progression and transplantation needs.
www.jhltopen.org
December 5, 2024 at 2:36 PM
CFTR gene cloned in 1989. Combined CFTR modulators elexacaftor-tezacaftor-ivacaftor (ETI) approved in 2019 -->
"The introduction of ETI has dramatically reduced the need for lung transplants among people with CF, reflecting significant improvements in lung function and disease management."🧪⚕️
"The introduction of ETI has dramatically reduced the need for lung transplants among people with CF, reflecting significant improvements in lung function and disease management."🧪⚕️
Today, I'm finally putting the Ra's al Ghul pronunciation debate to sleep once and for all.
www.youtube.com/watch?v=CfTR...
www.youtube.com/watch?v=CfTR...
The Problem With Ra's al Ghul
YouTube video by Comic Drake
www.youtube.com
August 22, 2026 at 4:18 PM
Today, I'm finally putting the Ra's al Ghul pronunciation debate to sleep once and for all.
www.youtube.com/watch?v=CfTR...
www.youtube.com/watch?v=CfTR...
ironically his daughter benefits from groundbreaking NIH-supported research (including by former NIH director Francis Collins) that led to discovery of the mutated gene (CFTR) that causes cystic fibrosis (CF) & development of lifesaving CF therapies
this is a psychic conflict, not a moral one
this is a psychic conflict, not a moral one
April 5, 2026 at 2:20 AM
ironically his daughter benefits from groundbreaking NIH-supported research (including by former NIH director Francis Collins) that led to discovery of the mutated gene (CFTR) that causes cystic fibrosis (CF) & development of lifesaving CF therapies
this is a psychic conflict, not a moral one
this is a psychic conflict, not a moral one
Happy to have this work out! Using cryo-EM and electrophysiology, we clarify the mechanism of a small molecule inhibitor of CFTR that could have some important clinical applications.
rdcu.be/eyVyq
rdcu.be/eyVyq
Structure of CFTR bound to (R)-BPO-27 unveils a pore-blockage mechanism
Nature Communications - Hyperactivation of the cystic fibrosis transmembrane conductance regulator (CFTR) is central to the pathogenesis of secretory diarrheas and autosomal dominant polycystic...
rdcu.be
August 2, 2025 at 4:31 PM
Happy to have this work out! Using cryo-EM and electrophysiology, we clarify the mechanism of a small molecule inhibitor of CFTR that could have some important clinical applications.
rdcu.be/eyVyq
rdcu.be/eyVyq
I sobbed when I was reading about the outcomes last year. Another good happy cry? The treatment of cystic fibrosis. www.childrenscolorado.org/advances-ans... Simply incredible. I had a med tech tell me her husband was getting this a year ago and I thought she was delusional it is so amazing.
Revolutionizing Cystic Fibrosis Care
New CFTR modulator treatment is a game changer
www.childrenscolorado.org
February 8, 2025 at 2:36 AM
I sobbed when I was reading about the outcomes last year. Another good happy cry? The treatment of cystic fibrosis. www.childrenscolorado.org/advances-ans... Simply incredible. I had a med tech tell me her husband was getting this a year ago and I thought she was delusional it is so amazing.
Issue 11 is complete!
Cover: Differentiating CFTR high expresser cells (CHEs) & their likely progenitors in rat intestinal jejunum. Notch2 (white) in crypt progenitors & differentiated villar CHEs, Meis1 (magenta) & CFTR (yellow) along crypt-villus axis. See Zagoren et al:
doi.org/10.1242/dev....
Cover: Differentiating CFTR high expresser cells (CHEs) & their likely progenitors in rat intestinal jejunum. Notch2 (white) in crypt progenitors & differentiated villar CHEs, Meis1 (magenta) & CFTR (yellow) along crypt-villus axis. See Zagoren et al:
doi.org/10.1242/dev....
June 16, 2025 at 3:11 PM
Issue 11 is complete!
Cover: Differentiating CFTR high expresser cells (CHEs) & their likely progenitors in rat intestinal jejunum. Notch2 (white) in crypt progenitors & differentiated villar CHEs, Meis1 (magenta) & CFTR (yellow) along crypt-villus axis. See Zagoren et al:
doi.org/10.1242/dev....
Cover: Differentiating CFTR high expresser cells (CHEs) & their likely progenitors in rat intestinal jejunum. Notch2 (white) in crypt progenitors & differentiated villar CHEs, Meis1 (magenta) & CFTR (yellow) along crypt-villus axis. See Zagoren et al:
doi.org/10.1242/dev....
Have you ever heard about Cif - a bacterial CFTR inhibitory factor? Yes? And have you heard that it goest beyond cystic fibrosis? And even beyond humans?
At ISCB-LATAM SoIBio SPBBC 2026, I'll tell about this in my virtual talk and poster:
www.iscb.org/latam2026/
🧪🦠🌾
@iscb.bsky.social
#LATAM2026
At ISCB-LATAM SoIBio SPBBC 2026, I'll tell about this in my virtual talk and poster:
www.iscb.org/latam2026/
🧪🦠🌾
@iscb.bsky.social
#LATAM2026
September 21, 2026 at 6:05 PM
Have you ever heard about Cif - a bacterial CFTR inhibitory factor? Yes? And have you heard that it goest beyond cystic fibrosis? And even beyond humans?
At ISCB-LATAM SoIBio SPBBC 2026, I'll tell about this in my virtual talk and poster:
www.iscb.org/latam2026/
🧪🦠🌾
@iscb.bsky.social
#LATAM2026
At ISCB-LATAM SoIBio SPBBC 2026, I'll tell about this in my virtual talk and poster:
www.iscb.org/latam2026/
🧪🦠🌾
@iscb.bsky.social
#LATAM2026
I went down a rabbit hole starting with a bacterial toxin that inhibits CFTR 🧪
It led me to plant hormones, insect symbionts, oil biodegradation — and a rather strange hypothesis about how bacteria may switch hosts 🌾🦠🦋
Read the story behind this hypothesis:
oxylipins.substack.com/p/the-role-o...
It led me to plant hormones, insect symbionts, oil biodegradation — and a rather strange hypothesis about how bacteria may switch hosts 🌾🦠🦋
Read the story behind this hypothesis:
oxylipins.substack.com/p/the-role-o...
September 25, 2026 at 1:13 PM
I went down a rabbit hole starting with a bacterial toxin that inhibits CFTR 🧪
It led me to plant hormones, insect symbionts, oil biodegradation — and a rather strange hypothesis about how bacteria may switch hosts 🌾🦠🦋
Read the story behind this hypothesis:
oxylipins.substack.com/p/the-role-o...
It led me to plant hormones, insect symbionts, oil biodegradation — and a rather strange hypothesis about how bacteria may switch hosts 🌾🦠🦋
Read the story behind this hypothesis:
oxylipins.substack.com/p/the-role-o...
An optimized adenine base editing strategy can repair the “untreatable” 1717-1G>A #CysticFibrosis mutation in cells and organoids with potentially promising efficiency rates and little off-target editing, according to new work in #ScienceTranslationalMedicine. https://scim.ag/4cvaqDk
Functional correction of the untreatable CFTR 1717-1G>A mutation through mRNA- and sgRNA-optimized base editing
An RNA-delivered base editing strategy efficiently corrects the CFTR 1717-1G>A mutation that is otherwise unresponsive to current drug therapies.
scim.ag
April 27, 2026 at 6:00 PM
An optimized adenine base editing strategy can repair the “untreatable” 1717-1G>A #CysticFibrosis mutation in cells and organoids with potentially promising efficiency rates and little off-target editing, according to new work in #ScienceTranslationalMedicine. https://scim.ag/4cvaqDk
A new class of drugs called #CFTR modulators (like Trikafta) are extending the lives of people with #cysticfibrosis, but some folks struggle with psychological side effects, and that deserves investigation. journals.lww.com/co-pulmonary...
Neuropsychiatric adverse effects from CFTR modulators... : Current Opinion in Pulmonary Medicine
findings
Studies show that a minority of persons with cystic fibrosis (PwCF) initiating cystic fibrosis transmembrane conductance regulator (CFTR) modulators experience neuropsychiatric AEs includin...
journals.lww.com
September 14, 2023 at 2:39 AM
A new class of drugs called #CFTR modulators (like Trikafta) are extending the lives of people with #cysticfibrosis, but some folks struggle with psychological side effects, and that deserves investigation. journals.lww.com/co-pulmonary...
🚨 Happy to share our first pre-print 🎉 on the causes of ribosome-associated degradation of CFTR and other transmembrane proteins.
We explore how protein folding, ER insertion, and elongation dynamics influence translation arrests in this new class of RQC targets.👇
www.biorxiv.org/content/10.1...
We explore how protein folding, ER insertion, and elongation dynamics influence translation arrests in this new class of RQC targets.👇
www.biorxiv.org/content/10.1...
Principles of ribosome-associated protein quality control during the synthesis of CFTR
Prolonged translational arrests caused by defective mRNAs activate the ribosome-associated protein quality control (RQC) pathway, which marks harmful incomplete proteins for degradation. Multipass tra...
www.biorxiv.org
August 1, 2025 at 10:36 AM
🚨 Happy to share our first pre-print 🎉 on the causes of ribosome-associated degradation of CFTR and other transmembrane proteins.
We explore how protein folding, ER insertion, and elongation dynamics influence translation arrests in this new class of RQC targets.👇
www.biorxiv.org/content/10.1...
We explore how protein folding, ER insertion, and elongation dynamics influence translation arrests in this new class of RQC targets.👇
www.biorxiv.org/content/10.1...
CFTR negatively reprograms Th2 cell responses and CFTR potentiation restrains allergic airway inflammation
insight.jci.org/articles/vie...
@jci-insight.bsky.social
insight.jci.org/articles/vie...
@jci-insight.bsky.social
March 25, 2025 at 4:48 PM
CFTR negatively reprograms Th2 cell responses and CFTR potentiation restrains allergic airway inflammation
insight.jci.org/articles/vie...
@jci-insight.bsky.social
insight.jci.org/articles/vie...
@jci-insight.bsky.social
And not merely by NIH but by former NIH Director Francis Collins, whose group cloned CFTR, defining the mechanistic basis for almost everything that followed in diagnosis and treatment.
April 30, 2025 at 5:26 PM
And not merely by NIH but by former NIH Director Francis Collins, whose group cloned CFTR, defining the mechanistic basis for almost everything that followed in diagnosis and treatment.
ok, mine is CFTR, obviously
September 16, 2026 at 5:58 PM
ok, mine is CFTR, obviously