Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

71 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood.
Hunanyan AS, Kantor B, Puranam RS, Elliott C, McCall A, Dhindsa J, Pagadala P, Wallace K, Poe J, Gunduz T, Asokan A, Koeberl DD, ElMallah MK, Mikati MA. Hunanyan AS, et al. Among authors: elmallah mk. Hum Gene Ther. 2021 Apr;32(7-8):405-419. doi: 10.1089/hum.2020.191. Epub 2021 Feb 12. Hum Gene Ther. 2021. PMID: 33577387 Free PMC article.
What's new and what's next for gene therapy in Pompe disease?
Roger AL, Sethi R, Huston ML, Scarrow E, Bao-Dai J, Lai E, Biswas DD, El Haddad L, Strickland LM, Kishnani PS, ElMallah MK. Roger AL, et al. Among authors: elmallah mk. Expert Opin Biol Ther. 2022 Sep;22(9):1117-1135. doi: 10.1080/14712598.2022.2067476. Epub 2022 Apr 27. Expert Opin Biol Ther. 2022. PMID: 35428407 Free PMC article.
Breathing in Duchenne muscular dystrophy: translation to therapy.
Mhandire DZ, Burns DP, Roger AL, O'Halloran KD, ElMallah MK. Mhandire DZ, et al. Among authors: elmallah mk. J Physiol. 2022 Aug;600(15):3465-3482. doi: 10.1113/JP281671. Epub 2022 Jun 24. J Physiol. 2022. PMID: 35620971 Free PMC article. Review.
GAA deficiency disrupts distal airway cells in Pompe disease.
El Haddad L, Lai E, Murthy PKL, Biswas DD, Soufny R, Roger AL, Tata PR, ElMallah MK. El Haddad L, et al. Among authors: elmallah mk. Am J Physiol Lung Cell Mol Physiol. 2023 Sep 1;325(3):L288-L298. doi: 10.1152/ajplung.00032.2023. Epub 2023 Jun 27. Am J Physiol Lung Cell Mol Physiol. 2023. PMID: 37366541
Pompe disease gene therapy.
Byrne BJ, Falk DJ, Pacak CA, Nayak S, Herzog RW, Elder ME, Collins SW, Conlon TJ, Clement N, Cleaver BD, Cloutier DA, Porvasnik SL, Islam S, Elmallah MK, Martin A, Smith BK, Fuller DD, Lawson LA, Mah CS. Byrne BJ, et al. Among authors: elmallah mk. Hum Mol Genet. 2011 Apr 15;20(R1):R61-8. doi: 10.1093/hmg/ddr174. Epub 2011 Apr 25. Hum Mol Genet. 2011. PMID: 21518733 Free PMC article. Review.
71 results