Delivering base editors to the liver and lungs in alpha-1 antitrypsin deficiency

  • Research Briefing
  • Published:

Nature Biotechnology

(2025)Cite this article

Subjects

We developed Dual Selective Organ-Targeting Lipid Nanoparticles (Dual SORT LNPs) capable of delivering base editors to multiple organs. Base editor Dual SORT LNPs corrected disease-causing mutations in both the liver and lungs, the primary organs affected in alpha-1 antitrypsin deficiency.

This is a preview of subscription content, access via your institution

Access options

Access Nature and 54 other Nature Portfolio journals

Get Nature+, our best-value online-access subscription

27,99 € / 30 days

cancel any time

Subscribe to this journal

Receive 12 print issues and online access

195,33 € per year

only 16,28 € per issue

Buy this article

  • Purchase on SpringerLink
  • Instant access to full article PDF

Prices may be subject to local taxes which are calculated during checkout

Fig. 1: Dual SORT LNPs effectively addressed toxicities in both the liver and lungs.

References

  1. Strnad, P., McElvaney, N. G. & Lomas, D. A. Alpha1-antitrypsin deficiency. N. Engl. J. Med. 382, 1443–1455 (2020). A review article on AATD.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  2. Zamora, M. R. & Ataya, A. Lung and liver transplantation in patients with alpha-1 antitrypsin deficiency. Ther. Adv. Chronic Dis. 12, (Suppl.) 20406223211002988 (2021). A review article that presents current treatment options for AATD.

  3. Stiles, K. M. et al. Intrapleural gene therapy for alpha-1 antitrypsin deficiency-related lung disease. Chronic Obstr. Pulm. Dis. 5, 244–257 (2018). A review article on gene therapy for AATD.

    PubMed 
    PubMed Central 

    Google Scholar
     

  4. Cheng, Q. et al. Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-Cas gene editing. Nat. Nanotechnol. 15, 313–320 (2020). This paper reports the LNP SORT methodology.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  5. Beam Therapeutics. A phase 1/2 dose-exploration and dose-expansion study to evaluate the safety and efficacy of BEAM-302 in adult patients with alpha-1 antitrypsin deficiency (AATD)-associated lung disease and/or liver disease. https://clinicaltrials.gov/study/NCT06389877 (2025). A clinical study that presents base editor AATD treatment.

Download references

Additional information

Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

This is a summary of: Kim, M. et al. Dual SORT LNPs for multi-organ base editing. Nat. Biotechnol. https://doi.org/10.1038/s41587-025-02675-z (2025).

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Delivering base editors to the liver and lungs in alpha-1 antitrypsin deficiency.
Nat Biotechnol (2025). https://doi.org/10.1038/s41587-025-02705-w

Download citation

  • Published:

  • DOI: https://doi.org/10.1038/s41587-025-02705-w

Read More

0 Votes: 0 Upvotes, 0 Downvotes (0 Points)

Leave a reply

Recent Comments

No comments to show.

Stay Informed With the Latest & Most Important News

I consent to receive newsletter via email. For further information, please review our Privacy Policy

Advertisement

Loading Next Post...
Follow
Sign In/Sign Up Sidebar Search Trending 0 Cart
Popular Now
Loading

Signing-in 3 seconds...

Signing-up 3 seconds...

Cart
Cart updating

ShopYour cart is currently is empty. You could visit our shop and start shopping.