Markus Y. Mapara 🇺🇦

Director Adult BMT&Cell Therapy Program at Columbia University Irving Medical Center/NYP. Passionate about #SickleCell Transplant and #GVHD. Tweets are mine.

[NEJM @NEJM]

Original Article: Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease (BEACON study) https://t.co/fdrUyAa6Ta

Editorial: More Options for Gene Editing in Hemoglobinopathies https://t.co/8amzRCFE0G

Further reading:
📄 Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease (RUBY study) https://t.co/d0rIzzotFU
📄 Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia (EdiThal study) https://t.co/rZUvlLzAvL

#Hematology #Genetics

[NEJM @NEJM]

Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease (RUBY study) https://t.co/d0rIzzotFU

Editorial: More Options for Gene Editing in Hemoglobinopathies https://t.co/8amzRCFE0G

Further reading:
📄 Original Article: Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease (BEACON study) https://t.co/fdrUyAa6Ta
📄 Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia (EdiThal study) https://t.co/rZUvlLzAvL

[NEJM @NEJM]

In the EdiThal study, a Cas12a–guide RNA complex was used to target the promoters of HBG1 and HBG2 in autologous stem cells to treat transfusion-dependent β-thalassemia. Full study results: https://t.co/rZUvlLzAvL

Editorial: More Options for Gene Editing in Hemoglobinopathies https://t.co/8amzRCFE0G

Further reading:
📄 Original Article: Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease (BEACON study) https://t.co/fdrUyAa6Ta
📄 Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease (RUBY study) https://t.co/d0rIzzotFU

[NEJM @NEJM]

In the RUBY study, a Cas12a–guide RNA complex was used to target the promoters of HBG1 and HBG2 in autologous stem cells to treat sickle cell disease. Full study results: https://t.co/d0rIzzotFU

Editorial: More Options for Gene Editing in Hemoglobinopathies https://t.co/8amzRCFE0G

Further reading:
📄 Original Article: Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease (BEACON study) https://t.co/fdrUyAa6Ta
📄 Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia (EdiThal study) https://t.co/rZUvlLzAvL

[NEJM @NEJM]

In the BEACON study involving persons with sickle cell disease, adenine base editing was used to target the promoters of HBG1 and HBG2 in autologous hematopoietic stem cells to increase fetal hemoglobin expression. Full study results: https://t.co/fdrUyAa6Ta

Editorial: More Options for Gene Editing in Hemoglobinopathies https://t.co/8amzRCFE0G

Further reading:
📄 Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease (RUBY study) https://t.co/d0rIzzotFU
📄 Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia (EdiThal study) https://t.co/rZUvlLzAvL

[Markus Y. Mapara 🇺🇦 @BMT_updates]

Wonderful essay by ⁦@SLentzsch⁩ and Paul Mapara just published in ⁦@TheFP⁩ ⁦@ElishaWiesel⁩ #HolocaustMemorialday https://t.co/CFTe4Nr56y

[Starzl Transplantation Institute @InstituteStarzl]

TODAY‼️Be there or be square🤓

[Shubhendu Trivedi @_onionesque]

Eliza O'Reilly has an excellent (developing) body of work on randomized decision trees and forests. Here is one of the earlier works.
https://t.co/gCFTFmTknL

[Markus Y. Mapara 🇺🇦 @BMT_updates]

🚨 new⁦@ASH_hematology heme talks⁩ podcast with Dr Shalini Shenoy ⁦@WUSTL⁩ on BMT and 🧬 therapy options in Sickle Cell Disease. ⁦@nyphospital⁩ ⁦@Columbia⁩ https://t.co/o4GnDrEF9b

[Michael Hughes @MSHughes_MD]

@JingFu_PhD presenting fascinating data from the @SLentzsch and @BMT_updates Laboratories on the production and in vitro/in vivo performance of #ALamyloidosis targeted CAR-phagocytes!!! Game changing antifibril therapies in development @columbiacancer

[German Embassy Washington @GermanyinUSA]

#OnThisDay in 1989, the Berlin Wall fell, marking the end of a divided Germany. 35 years later, we remember the brave citizens of the former GDR who stood up for their freedom. We also remember the critical role that the US played in German Reunification. Thank you, USA! 🇩🇪🇺🇸

[Sickle Cell 101 @sicklecell101]

Have you ever thought about undergoing a bone marrow transplant (BMT) to treat #sicklecell disease (SCD)? 🤔Here are three facts you should know about BMT for SCD.

#SickleCell101 #BoneMarrowTransplant #SickleCellAwareness

[Shernan Holtan MD @sghmd]

🚨Breaking News/Good News from #ASH24🚨

>90% 1-year OS in PTCy-treated alloHCT recipients age 70+ enrolled in @BMTCTN 1703! 😱

Important analysis by @sameem_abedin @MediHumdani @GvHD_Meade and team!! 🙌

https://t.co/afM75mk4nk

#PTCyrules

[Markus Y. Mapara 🇺🇦 @BMT_updates]

The best way to deal with #Election2024 anxiety is by attending the @ASTCT 🧬 therapy summit in Boston and listening to a fascinating lecture by @harvardmed Dean George Daley on the promise of hematopoietic gene therapy and learning about the first example of cell therapy

[Markus Y. Mapara 🇺🇦 @BMT_updates]

@ASTCT @ASH_hematology @CCTI_NYC @nyphospital

[Columbia Daily Spectator @ColumbiaSpec]

Michel Sadelain, a global pioneer in CAR T-cell immunotherapy, will head the new Columbia Initiative in Cell Engineering and Therapy.
https://t.co/d4633i7Ptl

[Markus Y. Mapara 🇺🇦 @BMT_updates]

Very nice article about 🧬 therapy activities at @Columbia highlighting progress in ENT by Lawrence Lustig, our BMT team in #sicklecell , cardiology by @MathewMaurer and work by Sam Sternberg’s group. @nyphospital @ColumbiaMed

[Columbia Medicine @ColumbiaMed]

.@columbiaps faculty are pushing gene therapy into its next phase, successfully treating deafness, sickle cell disease, and cardiac amyloidosis. Learn more about their groundbreaking research in the Fall 2024 issue of Columbia Medicine Magazine.
https://t.co/ljwyt7RB3i

[Michael Hughes @MSHughes_MD]

Backstage at the Columbia University AL Amyloidosis Symposium, with the formidable lineup of @SLentzsch, @DivayaB, @rajshekharucms, @BMT_updates, Dr Radhakrishnan, and Dr Maurer! @columbiacancer #ALamyloidosis

[CAMP@CUIMC @CAMPamyloidosis]

Join us at @CAMPamyloidosis @columbiacancer for a comprehensive update on management if AL Amyloidosis!
@SLentzsch @MathewMaurer

[Markus Y. Mapara 🇺🇦 @BMT_updates]

Learn everything you need to know about AL-Amyloidosis with an all-star team
@SLentzsch @MathewMaurer @jradnephro @rajshekharucms @Columbia
@ASH_hematology @ASTCT @BMT_HP11 @Amyloidosisfdn @IMFmyeloma

[Columbia Med CME @CUIMCMedCME]

THIS WEEK: AL #Amyloidosis (11/1); NEXT WEEK: Update in #HospitalMedicine (11/8); Infections in Hospitalized Patients (11/15) and more... Virtual Programs with video access and ACCME Credits. Registration and info:
https://t.co/YKkWDnyevC

#Hospitalist #MedX #MedTwitter #InternalMedicine #InfectionPrevention #Nephrology