Chemical Biology and Therapeutics Science, Broad Institute of MIT and Harvard, Cambridge, MA, USA.; Department of Medicine, Harvard Medical School, Boston, MA, USA.; Divisions of Renal Medicine and Engineering, Brigham and Women's Hospital, Boston, MA, USA.
Soumyashree A Gangopadhyay studies the use of advanced gene-editing technologies, particularly CRISPR, to tackle high cholesterol levels. This condition is often a precursor to heart disease, which can lead to severe health issues like heart attacks. Gangopadhyay's research specifically involves using a technique that can modify genes responsible for cholesterol production in the liver. By delivering these gene-editing tools through tiny fat particles injected into the bloodstream, their work aims to provide a long-lasting solution that could reduce or eliminate the need for lifelong cholesterol medications.
Key findings
CRISPR-based treatment reduced cholesterol levels in monkeys by about 60% after a single injection.
The low cholesterol levels were maintained for at least 8 months without any further treatment.
This research highlights the potential of gene editing to offer transformative one-time treatments for heart disease patients.
Frequently asked questions
Does Dr. Gangopadhyay study cholesterol?
Yes, they focus specifically on using gene editing to lower cholesterol levels and its implications for heart disease.
What treatments has Dr. Gangopadhyay researched?
Dr. Gangopadhyay has researched CRISPR gene editing as a treatment to lower cholesterol permanently.
Is Dr. Gangopadhyay's work relevant to heart disease patients?
Yes, their research is highly relevant as it explores new ways to potentially eliminate the need for lifelong cholesterol medications in heart disease patients.
Publications in plain English
A general approach to identify cell-permeable and synthetic anti-CRISPR small molecules.
2022
Nature cell biology
Lim D, Zhou Q, Cox KJ, Law BK, Lee M +17 more
Plain English This study focused on finding better small molecules that can inhibit CRISPR-associated enzymes, which are used for gene editing. Researchers developed a new, efficient method to find these inhibitors that works with different types of enzymes and requires less of the enzyme to be effective. They discovered a new compound called BRD7586, which is twice as powerful as previous inhibitors and helps the CRISPR tool work more accurately, regardless of where it's used in the genome.
Who this helps: This helps researchers and doctors who use CRISPR technology for genetic treatments.
In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates.
2021
Nature
Musunuru K, Chadwick AC, Mizoguchi T, Garcia SP, DeNizio JE +37 more
Plain English Researchers used a gene-editing tool called CRISPR to permanently disable a gene in monkeys' livers that controls cholesterol production, delivering it through tiny fat particles injected into the bloodstream. After a single injection, the monkeys' cholesterol dropped by about 60% and stayed low for at least 8 months without any additional treatment. This proves that gene editing could offer heart disease patients a one-time treatment instead of taking cholesterol drugs for life.
Precision Control of CRISPR-Cas9 Using Small Molecules and Light.
2019
Biochemistry
Gangopadhyay SA, Cox KJ, Manna D, Lim D, Maji B +2 more
Plain English This research focuses on improving how CRISPR-Cas9 is used for gene editing by making its activity more precise. The scientists found that they could better control CRISPR-Cas9 by using small molecules and light to turn it on or off as needed, making it safer and more effective in applications like gene therapy. This matters because better control can reduce unwanted side effects, making treatments safer for patients.
Who this helps: This helps patients undergoing gene therapies and doctors using CRISPR for medical treatments.
A Singular System with Precise Dosing and Spatiotemporal Control of CRISPR-Cas9.
2019
Angewandte Chemie (International ed. in English)
Manna D, Maji B, Gangopadhyay SA, Cox KJ, Zhou Q +3 more
Plain English This research focused on improving the CRISPR-Cas9 gene-editing tool by creating a system that allows precise control over how much, when, and where it acts. The new system can quickly activate Cas9 with low-intensity light, avoids background activity when not in use, and is safe for living organisms. This is important because it enhances the reliability and effectiveness of gene editing, which can lead to better treatments in medicine.
Who this helps: This benefits researchers, doctors, and patients needing advanced genetic therapies.
A High-Throughput Platform to Identify Small-Molecule Inhibitors of CRISPR-Cas9.
2019
Cell
Maji B, Gangopadhyay SA, Lee M, Shi M, Wu P +13 more
Plain English This study focused on creating a new tool to control CRISPR-Cas9, a key technology used for editing genes. Researchers developed small molecules that can inhibit CRISPR-Cas9, which are lightweight, can easily enter cells, and work reliably in the body. They successfully identified these inhibitors and demonstrated the ability to control CRISPR-Cas9's activity, which is crucial for safely and effectively using gene-editing in research and therapy.
Who this helps: This helps researchers and doctors working on gene therapy and genome engineering.
Simultaneous Analysis of a Non-Lipidated Protein and Its Lipidated Counterpart: Enabling Quantitative Investigation of Protein Lipidation's Impact on Cellular Regulation.
2017
Analytical chemistry
Shala-Lawrence A, Blanden MJ, Krylova SM, Gangopadhyay SA, Beloborodov SS +2 more
Plain English This study introduces a new method called protein-lipidation quantitation (PLQ) to measure both the starting and finished forms of a process called protein lipidation, which affects how proteins regulate cellular functions. The researchers showed that PLQ works well even when the protein structure changes and confirmed a connection between a specific mutation in a protein called p53 and increased cellular lipidation activity. This finding helps us understand the complex roles that lipidation plays in cellular regulation and could inform future research or treatments related to that mutation.
Who this helps: This helps researchers and doctors studying how mutations affect cellular behavior and disease processes.
Simultaneous Site-Specific Dual Protein Labeling Using Protein Prenyltransferases.
2015
Bioconjugate chemistry
Zhang Y, Blanden MJ, Sudheer Ch, Gangopadhyay SA, Rashidian M +2 more
Plain English This study focused on improving how scientists label proteins, which is essential for many lab applications. Researchers tested different chemical compounds and found that a specific enzyme, called rat GGTase-I, was effective at recognizing larger, more complex molecules, allowing for the labeling of two different proteins at the same time. They successfully used this method to attach labels to a green fluorescent protein and a red fluorescent protein, highlighting how their method can create useful protein structures in a simple way.
Who this helps: This benefits researchers in biochemistry and molecular biology who need precise tools for studying proteins.
Targeted reengineering of protein geranylgeranyltransferase type I selectivity functionally implicates active-site residues in protein-substrate recognition.
2014
Biochemistry
Gangopadhyay SA, Losito EL, Hougland JL
Plain English This study focused on how a specific enzyme called protein geranylgeranyltransferase type I (GGTase-I) recognizes and modifies proteins. The researchers found that certain parts of the GGTase-I enzyme are crucial for determining which proteins it can attach to, and by altering these parts, they created new versions of GGTase-I that can choose different proteins to modify. This is important because it shows that enzymes can be designed to have different functions, which could help us better understand protein modifications and develop potential treatments for diseases related to these processes.
Who this helps: Patients with conditions linked to protein modifications and researchers studying these processes.
Expansion of protein farnesyltransferase specificity using "tunable" active site interactions: development of bioengineered prenylation pathways.
2012
The Journal of biological chemistry
Hougland JL, Gangopadhyay SA, Fierke CA
Plain English This study looked at how to change the way an enzyme called protein farnesyltransferase (FTase) interacts with different proteins in cells. Researchers found that by changing a few specific parts of the enzyme, they could create new versions with different, and sometimes very specific, abilities to attach lipids to proteins. This is important because it could lead to new methods for studying protein functions and creating customized pathways in cells to modify proteins as needed.
Who this helps: This benefits researchers and scientists working on protein functions and modifications, as well as potential applications in medical therapies.