17273688. BASE EDITING FOR TREATING HUTCHINSON-GILFORD PROGERIA SYNDROME simplified abstract (Vanderbilt University)
Contents
- 1 BASE EDITING FOR TREATING HUTCHINSON-GILFORD PROGERIA SYNDROME
- 1.1 Organization Name
- 1.2 Inventor(s)
- 1.3 BASE EDITING FOR TREATING HUTCHINSON-GILFORD PROGERIA SYNDROME - A simplified explanation of the abstract
- 1.4 Simplified Explanation
- 1.5 Potential Applications
- 1.6 Problems Solved
- 1.7 Benefits
- 1.8 Potential Commercial Applications
- 1.9 Possible Prior Art
- 1.10 Unanswered Questions
- 1.11 Original Abstract Submitted
BASE EDITING FOR TREATING HUTCHINSON-GILFORD PROGERIA SYNDROME
Organization Name
Inventor(s)
David R. Liu of Cambridge MA (US)
Luke W. Koblan of Cambridge MA (US)
Jonathan D. Brown of Nashville TN (US)
Charles Yang Lin of Houston TX (US)
BASE EDITING FOR TREATING HUTCHINSON-GILFORD PROGERIA SYNDROME - A simplified explanation of the abstract
This abstract first appeared for US patent application 17273688 titled 'BASE EDITING FOR TREATING HUTCHINSON-GILFORD PROGERIA SYNDROME
Simplified Explanation
The patent application discloses adenosine deaminases for treating Hutchinson-Gilford progeria syndrome (HGPS) by deaminating adenosine in DNA, as well as fusion proteins, guide RNAs, and compositions comprising a Cas9 domain and adenosine deaminases.
- Adenosine deaminases are capable of deaminating adenosine in DNA to treat Hutchinson-Gilford progeria syndrome (HGPS).
- Fusion proteins, guide RNAs, and compositions with a Cas9 domain and adenosine deaminases can correct mutations in genes like LMNA.
- The technology can be used to treat HGPS by correcting mutations in DNA.
- Adenosine deaminases can be used to correct the C1824T mutation in LMNA.
Potential Applications
The technology can be applied in gene therapy to treat genetic disorders like Hutchinson-Gilford progeria syndrome.
Problems Solved
This technology addresses the genetic mutations that cause Hutchinson-Gilford progeria syndrome, offering a potential treatment option for affected individuals.
Benefits
The use of adenosine deaminases and Cas9 technology can potentially correct genetic mutations and improve the health outcomes of individuals with HGPS.
Potential Commercial Applications
The technology could be commercialized for gene therapy treatments targeting genetic disorders like Hutchinson-Gilford progeria syndrome.
Possible Prior Art
Prior research may have explored the use of gene editing technologies like CRISPR-Cas9 for correcting genetic mutations associated with various diseases, including progeria.
Unanswered Questions
How effective is this technology in correcting genetic mutations in other diseases besides HGPS?
The effectiveness of this technology in correcting mutations in other genetic disorders remains to be fully understood and may require further research and clinical trials.
What are the potential long-term effects of using adenosine deaminases and Cas9 technology for gene therapy?
The long-term effects of utilizing this technology for gene therapy, including any off-target effects or unintended consequences, need to be thoroughly investigated to ensure its safety and efficacy in clinical applications.
Original Abstract Submitted
The disclosure provides adenosine deaminases that are capable of deaminating adenosine in DNA to treat Hutchin-son-Gilford progeria syndrome (HOPS). The disclosure also provides fusion proteins, guide RNAs and compositions comprising a Cas9 (e.g., a Cas9 nickase) domain and adenosine deaminases that deaminate adenosine in DNA, for example in a LNA gene. In some embodiments, adenosine deaminases provided herein are used to correct a C1824T mutation in LMNA. In some embodiments, the methods and compositions provided herein are used to treat Hutchinson-Gilford progeria syndrome (HGPS).
