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Mashup Score: 1Editing the Question on Gene Therapy from Can We to When Should We? - 1 month(s) ago
Editing the Question on Gene Therapy from Can We to When Should We? This is a continuation of sorts from my prior post here: “Time for a Revision on Gene Editing for Cholesterol?” One of my heroes in science is Linus Pauling, winner of two unshared Nobel Prizes. He was awarded the 1954 Nobel Prize in Chemistry in part for his work on sickle cell anemia, defining it as a molecular disease (
Source: venkmurthy.substack.comCategories: General Medicine News, CardiologistsTweet
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Mashup Score: 1Editing the Question on Gene Therapy from Can We to When Should We? - 5 month(s) ago
Editing the Question on Gene Therapy from Can We to When Should We? This is a continuation of sorts from my prior post here: “Time for a Revision on Gene Editing for Cholesterol?” One of my heroes in science is Linus Pauling, winner of two unshared Nobel Prizes. He was awarded the 1954 Nobel Prize in Chemistry in part for his work on sickle cell anemia, defining it as a molecular disease (
Source: venkmurthy.substack.comCategories: General Medicine News, CardiologistsTweet
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Mashup Score: 99Time for a Revision on Gene Editing for Cholesterol? - 6 month(s) ago
One of several highly anticipated clinical trials presented at the 2023 American Heart Association meeting was the heart-1 study (https://classic.clinicaltrials.gov/ct2/show/NCT05398029) from Verve Therapeutics. Some leading voices have hailed this a “game-changer” and the dawn of a new era. This was a small study of 10 individuals with heterozygous familial cholesterolemia (HeFH) – a genetic disorder affecting ~1 million Americans which causes elevated low-density lipoproteins (LDL or “bad cholesterol”). Patients with this condition often experience heart attacks, strokes and die prematurely. Fortunately, we have many treatment options available to mitigate (but not completely eliminate) the increased risks these patients experience including at least three types of oral medications (statin drugs are the mainstay) and several injectable options which can be added on. Three injectable drugs target a protein called PCSK9 which is involved in the processing of LDL. Individuals who lack P
Source: venkmurthy.substack.comCategories: General Medicine News, CardiologistsTweet
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Mashup Score: 97Time for a Revision on Gene Editing for Cholesterol? - 6 month(s) ago
One of several highly anticipated clinical trials presented at the 2023 American Heart Association meeting was the heart-1 study (https://classic.clinicaltrials.gov/ct2/show/NCT05398029) from Verve Therapeutics. Some leading voices have hailed this a “game-changer” and the dawn of a new era. This was a small study of 10 individuals with heterozygous familial cholesterolemia (HeFH) – a genetic disorder affecting ~1 million Americans which causes elevated low-density lipoproteins (LDL or “bad cholesterol”). Patients with this condition often experience heart attacks, strokes and die prematurely. Fortunately, we have many treatment options available to mitigate (but not completely eliminate) the increased risks these patients experience including at least three types of oral medications (statin drugs are the mainstay) and several injectable options which can be added on. Three injectable drugs target a protein called PCSK9 which is involved in the processing of LDL. Individuals who lack P
Source: venkmurthy.substack.comCategories: General Medicine News, CardiologistsTweet
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Mashup Score: 1Editing the Question on Gene Therapy from Can We to When Should We? - 6 month(s) ago
Editing the Question on Gene Therapy from Can We to When Should We? This is a continuation of sorts from my prior post here: “Time for a Revision on Gene Editing for Cholesterol?” One of my heroes in science is Linus Pauling, winner of two unshared Nobel Prizes. He was awarded the 1954 Nobel Prize in Chemistry in part for his work on sickle cell anemia, defining it as a molecular disease (
Source: venkmurthy.substack.comCategories: General Medicine News, CardiologistsTweet
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Mashup Score: 1Editing the Question on Gene Therapy from Can We to When Should We? - 6 month(s) ago
Editing the Question on Gene Therapy from Can We to When Should We? This is a continuation of sorts from my prior post here: “Time for a Revision on Gene Editing for Cholesterol?” One of my heroes in science is Linus Pauling, winner of two unshared Nobel Prizes. He was awarded the 1954 Nobel Prize in Chemistry in part for his work on sickle cell anemia, defining it as a molecular disease (
Source: venkmurthy.substack.comCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 95Time for a Revision on Gene Editing for Cholesterol? - 6 month(s) ago
One of several highly anticipated clinical trials presented at the 2023 American Heart Association meeting was the heart-1 study (https://classic.clinicaltrials.gov/ct2/show/NCT05398029) from Verve Therapeutics. Some leading voices have hailed this a “game-changer” and the dawn of a new era. This was a small study of 10 individuals with heterozygous familial cholesterolemia (HeFH) – a genetic disorder affecting ~1 million Americans which causes elevated low-density lipoproteins (LDL or “bad cholesterol”). Patients with this condition often experience heart attacks, strokes and die prematurely. Fortunately, we have many treatment options available to mitigate (but not completely eliminate) the increased risks these patients experience including at least three types of oral medications (statin drugs are the mainstay) and several injectable options which can be added on. Three injectable drugs target a protein called PCSK9 which is involved in the processing of LDL. Individuals who lack P
Source: venkmurthy.substack.comCategories: General Medicine News, CardiologistsTweet
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Mashup Score: 95Time for a Revision on Gene Editing for Cholesterol? - 6 month(s) ago
One of several highly anticipated clinical trials presented at the 2023 American Heart Association meeting was the heart-1 study (https://classic.clinicaltrials.gov/ct2/show/NCT05398029) from Verve Therapeutics. Some leading voices have hailed this a “game-changer” and the dawn of a new era. This was a small study of 10 individuals with heterozygous familial cholesterolemia (HeFH) – a genetic disorder affecting ~1 million Americans which causes elevated low-density lipoproteins (LDL or “bad cholesterol”). Patients with this condition often experience heart attacks, strokes and die prematurely. Fortunately, we have many treatment options available to mitigate (but not completely eliminate) the increased risks these patients experience including at least three types of oral medications (statin drugs are the mainstay) and several injectable options which can be added on. Three injectable drugs target a protein called PCSK9 which is involved in the processing of LDL. Individuals who lack P
Source: venkmurthy.substack.comCategories: General Medicine News, CardiologistsTweet
Link to my post on why although this huge advance for sickle cell patients, it perhaps isn't the time to use this in disease we have a lot of existing treatments for like heterozygous familial hypercholesterolemia https://t.co/5mBdhUIsPa