|Articles|April 15, 2002

Gene therapy breakthrough seen in Leber's congenital amaurosis

Philadelphia-Much progress has been made in the development of gene therapies for retinal dystrophies during the last decade. Albert Maguire, MD

Newsletter

Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Subscribe Now!

Related Articles

Latest CME

New Horizons in Multiple Myeloma Treatment – Selection and Sequencing From Newly Diagnosed to Relapsed/Refractory Disease
Video

New Horizons in Multiple Myeloma Treatment – Selection and Sequencing From Newly Diagnosed to Relapsed/Refractory Disease

Amrita Krishnan, MD; Suzanne Lentzsch, MD, PhD; Sagar Lonial, MD, FACP

View more
Exploring the Dynamic Shifts in the MDS Treatment Paradigm: Unveiling Advances in Disease Modifying Therapies, Spotlight on Cutting-Edge Clinical Trials, and the Latest Approvals
Multimedia

Exploring the Dynamic Shifts in the MDS Treatment Paradigm: Unveiling Advances in Disease Modifying Therapies, Spotlight on Cutting-Edge Clinical Trials, and the Latest Approvals

Amer Zeidan, MBBS, MHS; Michael R. Savona, MD

View more
Medical Crossfire®: Uncovering The Clinical Utility of Combination Therapies and MRD Assessment in Newly Diagnosed Multiple Myeloma
Video

Medical Crossfire®: Uncovering The Clinical Utility of Combination Therapies and MRD Assessment in Newly Diagnosed Multiple Myeloma

Ajai Chari, MD; Adriana Rossi, MD; Anupama Kumar, MD

View more
BURST Expert Illustrations and Commentaries™: Exploring the Mechanistic Rationale for CSF-1R– Directed Treatment in Chronic GVHD
Multimedia

BURST Expert Illustrations and Commentaries™: Exploring the Mechanistic Rationale for CSF-1R– Directed Treatment in Chronic GVHD

Corey Cutler, MD, MPH, FRCP(C); Carrie L. Kitko, MD

View more
Community Practice Connections™: 8th Annual School of Nursing Oncology™
Multimedia

Community Practice Connections™: 8th Annual School of Nursing Oncology™

Beth Sandy, MSN, CRNP, FAPO; Laura Wood, RN, MSN, OCN; Michael Lai, ARNP; Tiffany Richards, PhD, ANP-BC, AOCNP®; Lauren Verity Moore, DNP, MSN, AGACNP-BC; Megan Wanca, APRN, DNP, FNP-C

View more
SimulatEd™: Patient-Centered Care to Improve Outcomes in Chronic Lymphocytic Leukemia
Case-based Simulation

SimulatEd™: Patient-Centered Care to Improve Outcomes in Chronic Lymphocytic Leukemia

Catherine C. Coombs, MD

View more
(COPE) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care
Video

(COPE) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

Asim V. Farooq, MD; Joann Kang, MD; Nita Lee, MD, MPH

View more
(CME) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care
Video

(CME) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

Asim V. Farooq, MD; Joann Kang, MD; Nita Lee, MD, MPH

View more
Community Practice Connections™: 6th Annual Precision Medicine Symposium – An Illustrated Tumor Board
Multimedia

Community Practice Connections™: 6th Annual Precision Medicine Symposium – An Illustrated Tumor Board

Andre H. Goy, MD; Monique Hartley-Brown, MD, MMSc (Guest Co-Chair); Chiara Battelli, MD, PhD; Marios Giannakis, MD, PhD; Shail Maingi, MD; Aparna Parikh, MD

View more
Addressing Pivotal Updates in AML – Expert Perspectives on Individualizing Management for Special Populations
Video

Addressing Pivotal Updates in AML – Expert Perspectives on Individualizing Management for Special Populations

Eytan M. Stein, MD; Marina Kremyanskaya, MD, PhD; Hannah Levavi, MD

View more
Innovations in MDS Care: Navigating New Treatment Paradigms, Breakthrough Therapies, and Emerging Clinical Evidence
Video

Innovations in MDS Care: Navigating New Treatment Paradigms, Breakthrough Therapies, and Emerging Clinical Evidence

Rami Komrokji, MD; Maen Hussein, MD

View more
Annual Hematology Meeting: Preceding the 67th ASH Annual Meeting and Exposition
In-Person + Virtual Event

Annual Hematology Meeting: Preceding the 67th ASH Annual Meeting and Exposition

December 5, 2025

View more
Addressing Racial Disparities in Multiple Myeloma: Forging a Path Towards Health Equity in Clinical Trials and Everyday Practice
Video

Addressing Racial Disparities in Multiple Myeloma: Forging a Path Towards Health Equity in Clinical Trials and Everyday Practice

Craig Emmitt Cole, MD; Sikander Ailawadhi, MD; Irene Ghobrial, MD; Monique Hartley-Brown, MD, MMSc

View more
Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment
Multimedia

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

Elias Jabbour, MD

View more
How We Do It®: New Combinations Reshaping Treatment Decision-Making in Newly Diagnosed Multiple Myeloma
Video

How We Do It®: New Combinations Reshaping Treatment Decision-Making in Newly Diagnosed Multiple Myeloma

C. Ola Landgren, MD, PhD; Cristina Gasparetto, MD; Katja Weisel, MD

View more
Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Ever-Evolving Treatment of MF, PV, and ET
Video

Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Ever-Evolving Treatment of MF, PV, and ET

Rami S. Komrokji, MD; Ghaith Abu-Zeinah, MD; Gabriela Hobbs, MD; Barbara Mora, MD

View more
From Barriers to Breakthroughs in Multiple Myeloma: How Trials Compare to the Real-World Management of Patients in Your Practice
Video

From Barriers to Breakthroughs in Multiple Myeloma: How Trials Compare to the Real-World Management of Patients in Your Practice

Krina Patel, MD, MSc; Alfred L. Garfall, MD; Ajay K. Nooka, MD, MPH, FACP

View more
Show Me the Data: How Do We Navigate the Latest Evidence on Novel Therapies and Trials Across GvHD Prophylaxis, Acute, and Chronic Care?
Video

Show Me the Data: How Do We Navigate the Latest Evidence on Novel Therapies and Trials Across GvHD Prophylaxis, Acute, and Chronic Care?

Corey Cutler, MD, MPH, FRCPC; Amin M. Alousi, MD; Yi-Bin Chen, MD; Carrie Lynn Kitko, MD

View more
Show Me the Data™: Pushing CAR Ts Forward Across Lymphoid Malignancies…Accelerating Access in the Community and the Treatment Strategies for the Road Ahead
Video

Show Me the Data™: Pushing CAR Ts Forward Across Lymphoid Malignancies…Accelerating Access in the Community and the Treatment Strategies for the Road Ahead

Alexey V. Danilov, MD, PhD; Leidy L. Isenalumhe, MD, MS; Lori A. Leslie, MD; Mazyar Shadman, MD, MPH; Jason R. Westin, MD, MS, FACP

View more
Medical Crossfire®: New Horizons in Acute Lymphoblastic Leukemia—How The Experts Apply New Evidence from AYA to Adult Scenarios
Video

Medical Crossfire®: New Horizons in Acute Lymphoblastic Leukemia—How The Experts Apply New Evidence from AYA to Adult Scenarios

Elias Jabbour, MD; Hagop M. Kantarjian, MD; Aaron Logan, MD, PhD, MPhil; Rob Pieters, MD, PhD; Claire Roddie, MD, PhD

View more
SimulatEd™ in Multiple Myeloma: Personalizing Care Plans to Treat Newly Diagnosed Patients
Case-based Simulation

SimulatEd™ in Multiple Myeloma: Personalizing Care Plans to Treat Newly Diagnosed Patients

Amrita Krishnan, MD

View more
30th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma
In-Person Event

30th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma

February 26-28, 2026

Register now!
Medical Crossfire®: Expert Interpretations of the Latest Data in CLL Management – Understanding the Impact of Optimal Treatment Selection on Patient Outcomes
Video

Medical Crossfire®: Expert Interpretations of the Latest Data in CLL Management – Understanding the Impact of Optimal Treatment Selection on Patient Outcomes

Jennifer R. Brown, MD, PhD; Alexey V. Danilov, MD, PhD; Matthew S. Davids, MD, MMSc

View more
Community Practice Connections™: Tailored Treatment Approaches for Older Patients With Advanced HR+/HER2– Breast Cancer
Multimedia

Community Practice Connections™: Tailored Treatment Approaches for Older Patients With Advanced HR+/HER2– Breast Cancer

Hope S. Rugo, MD, FASCO; Neil M. Iyengar, MD; Komal Jhaveri, MD, FACP

View more
29th Annual International Congress on Hematologic Malignancies: Focus on Leukemias, Lymphomas, and Myeloma
Video

29th Annual International Congress on Hematologic Malignancies: Focus on Leukemias, Lymphomas, and Myeloma

André H. Goy, MD; Jorge E. Cortes, MD; Sagar Lonial, MD, FACP

View more
Cases & Conversations™: Transforming AML Care—Precision Strategies, Evolving Therapies, and Clinical Insights
Video

Cases & Conversations™: Transforming AML Care—Precision Strategies, Evolving Therapies, and Clinical Insights

Gail J. Roboz, MD; Hagop Kantarjian, MD; Catherine Lai, MD, MPH; Amer Zeidan, MBBS, MHS

View more
Medical Crossfire®: Harnessing the Power of Modern Therapies in Newly Diagnosed Multiple Myeloma
Video

Medical Crossfire®: Harnessing the Power of Modern Therapies in Newly Diagnosed Multiple Myeloma

C. Ola Landgren, MD, PhD; Mohamad Mohty, MD, PhD; Surbhi Sidana, MD

View more
Medical Crossfire®: Improving Patient Outcomes in Myeloproliferative Neoplasms With Novel Therapeutic Approaches
Video

Medical Crossfire®: Improving Patient Outcomes in Myeloproliferative Neoplasms With Novel Therapeutic Approaches

Rami Komrokji, MD; Gabriela Hobbs, MD; John Mascarenhas, MD; Naveen Pemmaraju, MD

View more
Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma
Video

Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma

C. Ola Landgren, MD, PhD; Mohamad Mohty, MD, PhD; Surbhi Sidana, MD

View more
10th Annual Live Medical Crossfire®: Hematologic Malignancies
In-Person Event

10th Annual Live Medical Crossfire®: Hematologic Malignancies

July 11, 2026

Register now!
Expert Perspectives on Applying Recent Evidence to Practice in the Management of Newly Diagnosed Multiple Myeloma
Video

Expert Perspectives on Applying Recent Evidence to Practice in the Management of Newly Diagnosed Multiple Myeloma

Amrita Krishnan, MD; Meletios Athanasios (Thanos) C. Dimopoulos, MD; Shaji Kumar, MD; Katja Weisel, MD

View more
Imaging Matters: Decoding PSMA PET for Better Decision-Making
Multimedia

Imaging Matters: Decoding PSMA PET for Better Decision-Making

Jeremie Calais, MD, PhD; Tanya B. Dorff, MD; Scott T. Tagawa, MD, MS, FACP, FASCO

View more
Go To PER in Chicago
In-Person + Virtual Event

Go To PER in Chicago

On-Demand Sessions Available Now!

View more
Contact Info

259 Prospect Plains Rd, Bldg H
Monroe, NJ 08831

609-716-7777

HCP Live
Brand Logo

© 2025 MJH Life Sciences®

All rights reserved.

Home
About Us
News
Contact Us